[Federal Register Volume 63, Number 63 (Thursday, April 2, 1998)]
[Rules and Regulations]
[Pages 16296-16338]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 98-8191]
[[Page 16295]]
_______________________________________________________________________
Part II
Department of Health and Human Services
_______________________________________________________________________
42 CFR Part 121
Organ Procurement and Transplantation Network; Final Rule
Federal Register / Vol. 63, No. 63 / Thursday, April 2, 1998 / Rules
and Regulations
[[Page 16296]]
DEPARTMENT OF HEALTH AND HUMAN SERVICES
42 CFR PART 121
[Docket Number: 98-HRSA-01]
RIN 0906-AA32
Organ Procurement and Transplantation Network
AGENCY: Health Resources and Services Administration, HHS.
ACTION: Final rule with comment period.
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SUMMARY: This document sets forth the final rule governing the
operation of the Organ Procurement and Transplantation Network (OPTN),
which performs a variety of functions related to organ transplantation
under contract with HHS. The document also offers a 60 day period for
additional public comment. The rule will become effective 30 days
following the close of the comment period. If the Department believes
that additional time is required to review the comments, we will
consider delaying the effective date. In combination with a new
National Organ and Tissue Donation Initiative, this rule is intended to
improve the effectiveness and equity of the Nation's transplantation
system and to further the purposes of the National Organ Transplant Act
of 1984, as amended. These purposes include: encouraging organ
donation; developing an organ allocation system that functions as much
as technologically feasible on a nationwide basis; providing the bases
for effective Federal oversight of the OPTN (as well as for
implementing related provisions in the Social Security Act); and,
providing better information about transplantation to patients,
families and health care providers.
DATES: These regulations are effective July 1, 1998.
Comments on this final rule are invited. To ensure consideration,
comments must be received by June 1, 1998.
ADDRESSES: Written comments should be addressed to Jon L. Nelson,
Associate Director, Office of Special Programs, Room 123, Park
Building, 12420 Parklawn Drive, Rockville, MD 20857. All comments
received will be available for public inspection and copying at the
above address, weekdays (Federal holidays excepted) between the hours
of 9:00 a.m. and 4:00 p.m. A copy of this rule, and selected background
materials, will be posted on the Division of Transplantation Internet
site at http://www.hrsa.dhhs.gov/bhrd/dot/dotmain.htm.
FOR FURTHER INFORMATION CONTACT: Jon L. Nelson, Associate Director,
Office of Special Programs, Room 7-29, Parklawn Building, 5600 Fishers
Lane, Rockville, MD 20857; telephone (301) 443-7577.
SUPPLEMENTARY INFORMATION: Over the past two decades, the safety and
survival rates for transplantation of human organs have improved
markedly, and the number of transplants has increased. In 1996, about
20,000 transplants were performed in the United States. At the same
time, the rapid development of transplant techniques and the growth of
the Nation's transplant system present new challenges:
1. The demand for organs for transplantation exceeds the supply,
and this gap is growing. About 4,000 persons died in 1996 while
awaiting transplantation.
2. The Nation's organ allocation system remains heavily weighted to
the local use of organs instead of making organs available on a broader
regional or national basis for patients with the greatest medical need
consistent with sound medical judgment. Technological advances have
made it possible to preserve organs longer and share them more widely,
but the allocation system does not yet take full advantage of this
capacity. Instead, some patients with less urgent medical need receive
transplants before other patients with greater medical need whether
listed locally or away from home.
3. The criteria used in listing those who need transplantation vary
from one transplant center to another, as do the criteria used to
determine the medical status of a patient. This lack of uniform,
medically objective criteria make it difficult to compare the medical
need of patients in different centers.
4. As a result of both the local preference in allocation and the
lack of standard medical criteria, waiting times for organs are much
longer in some geographic areas than in others. The statute envisions a
national allocation system, based on medial criteria, which results in
the equitable treatment of transplant patients. But equitable treatment
cannot be assured if medical criteria vary from one transplant center
to another and if allocation policies prevent suitable organs from
being offered first to those with the greatest medical need.
5. Useful, current, transplant-center specific data for patients
and health care providers are not available, despite information
technology advances that make more current reporting feasible.
Efforts are needed to address these challenges in the areas of both
donation and allocation:
In order to bring about substantial increases in the number of
organ donors and the number of transplants performed each year, a new
National Organ and Tissue Donation Initiative has been launched.
Working in partnership with national and local organizations, the
Department of Health and Human Services (HHS) seeks to increase
donation through encouraging more individuals to chose to be organ
donors and that share that decision with their families; through
improved performance by hospitals and organ procurement organizations
toward ensuring that the families of potential donors are given the
opportunity to allow donation; through higher consent rates by
families, especially by encouraging those who elect to be organ donors
to inform their families of their decision; and through new research on
enhancing donation. Proposed regulations affecting hospitals and organ
procurement organizations were published December 19, 1997 (62 FR
66725). The Department expects that the supply of organs may be raised
by about 20 percent through this initiative, which would greatly
alleviate organ shortages.
In order to improve allocation of organs for transplantation, this
final rule establishes performance goals to be achieved by the OPTN.
Actions already underway in the OPTN are consistent with several of
these goals. The rule does not establish specific allocation policies,
but instead looks to the organ transplant community to take action to
meet the performance goals. The goals include:
Minimum Listing Criteria--The OPTN is required to define
objective and measurable medical criteria to be used by all transplant
centers in determining whether a patient is appropriate to be listed
for a transplant. In this way, patients with essentially the same
medical need will be listed in the same way at all transplant centers.
Status Categories--The OPTN is required to determine
objective medical criteria to be used nationwide in determining the
medical status of those awaiting transplantation. This will provide a
common measurement for use by all transplant centers in determining the
urgency of an individual's medical condition, and it will facilitate
OPTN efforts to direct organs to those with greatest medical need, in
accordance with sound medical judgment.
Equitable Allocation--The OPTN is required to develop
equitable allocation policies that provide organs to those with the
greatest medical urgency, in accordance with sound medical judgment.
This increases the likelihood of patients obtaining matching organs,
[[Page 16297]]
and gives all patients equal chances to obtain organs compared to other
patients of equal medical status, wherever they live or list.
By requiring common criteria for listing eligibility and medical
status, and by requiring that organs be directed so as to equalize
waiting times, especially for those with greatest medical need, this
rule is designed to provide patients awaiting transplants with equal
access to organs and to provide organs to sickest patients first,
consistent with sound medical judgment. While present OPTN policies
give weight to medical need, the ``local first'' practice thwarts organ
allocation over a broad area and thus prevents medical need from being
the dominant factor in allocation decisions.
Under the provisions of this rule, it is intended that the area
where a person lives or the transplant center where he or she is listed
will not be primary factors in how quickly he or she receives a
transplant. Instead, organs will be allocated according to objective
standards of medical status and need. In this way, suitable organs will
reach patients with the greatest medical need, both when they are
procured locally and when they are procured outside the listed
patients' areas. This objective reflects the views of many commenters
on the proposed regulations, as well as the finding of the American
Medical Association in its Code of Medical Ethics: ``Organs should be
considered a national, rather than a local or regional resource.
Geographical priorities in the allocation of organs should be
prohibited except when transportation of organs would threaten their
suitability for transplantation.''
The OPTN is required to develop proposals for the new allocation
policies (except for livers) within a year of the effective date of the
final rule. In the case of liver allocation policies, where policy
development work has been underway for several years, the OPTN is
required to develop a new proposed allocation policy within 60 days of
the effective date.
Other provisions of this rule include requirements that the OPTN
make more current data available for the public, including measures of
performance of individually identified transplant centers. This
information is needed by patients, families, physicians, and payers in
choosing a course of action and is needed as a quality measurement
instrument.
In addition, the rule defines the governing structure of the OPTN
and outlines procedures for the establishment of policies by the OPTN
that include appropriate participation by transplant professionals and
families, with oversight by HHS. The rule also includes a requirement
that the OPTN develop a ``grandfathering'' proposal for patients
currently awaiting liver transplantation so that these patients are
treated no less favorably under the new allocation policies than they
would have been under current allocation policies. The OPTN also is
required to develop proposed transition policies for the initial
changes required by this rule to its allocation policies for other
organs.
The National Organ and Tissue Donation Initiative and this final
rule build on more than a decade of experience, including improving
medical technology, to create a national community of organ sharing and
to save and improve more lives through transplantation. The rule
defines Federal expectations, based on the role given to the Secretary
under the statute, but looks to the OPTN to propose policy choices that
meet those expectations.
The remainder of this preamble is arranged under the following
headings.
I Background
A. Overview
B. Legislative and Regulatory History
C. DHHS and OPTN Relationships
D. Enforcement
1. Section 1138 of the Social Security Act
2. OPTN Policies
II Summary of Public Comments and Policies of the Final Rule
A. Summary of Original Public Comments
B. Summary of Public Hearing
C. The Department's Response and Policies of the Final Rule
1. Sec. 121.2--Definitions
2. Sec. 121.3--The OPTN
3. Sec. 121.5--Listing Requirements
4. Sec. 121.6--Organ Procurement
5. Sec. 121.7--Identification of Organ Recipient
6. Sec. 121.4--Policies: Secretarial Review
7. Sec. 121.8--Allocation of Organs
(a) Indicator Data
(b) Deadlines (Sec. 121.8(c))
(c) Liver Allocation Policies
(d) Directed Donation (Sec. 121.7)
8. Sec. 121.9--Designated Transplant Program Requirements
9. Sec. 121.10--Reviews, Evaluation, and Enforcement
10. Sec. 121.4(d)--Appeals of OPTN Policies and Procedures
11. Sec. 121.11--Record Maintenance and Reporting Requirements
12. Sec. 121.12--Preemption
III Economic and Regulatory Impact
A. Legal Requirements
B. Effects of Organ Transplantation
C. Effects of this Rule
D. Alternatives Considered
E. Effects on Transplant Programs
IV Paperwork Reduction Act of 1995
I. Background
A. Overview
The National Organ Transplant Act of 1984 (NOTA) created the Organ
Procurement and Transplantation Network (OPTN). The Act has been the
subject of two major sets of amendments. In each instance, the Congress
acted to encourage the development of a fair, national system of organ
allocation. The original statute (Pub. L. 98-507, title II, Sec. 201,
formerly codified at 42 U.S.C. 274(b)(2)(C)) required the OPTN to
``assist organ procurement organizations in the distribution of organs
which cannot be placed within the service areas of the organizations.''
(Emphasis supplied.) However, the underscored language was removed in a
1988 amendment to the NOTA (Pub .L. 100-607, title IV, Sec. 403,
formerly codified at 42 U.S.C. 274(b)(2)(D)), according to the Senate
``so as to remove any statutory bias respecting the important question
of criteria for the proper distribution of organs among patients.'' S.
Rep. No. 100-310 at 14-15 (1988). In 1990, this language was again
rewritten, this time to require that the OPTN ``assist organ
procurement organizations in the nationwide distribution of organs
equitably among transplant patients.'' (Emphasis supplied.) Pub. L.
101-616, title II, Sec. 202, now codified at 42 U.S.C. 274(b)(2)(D).
The Senate explained that ``[b]ecause the demand for transplantable
organs is expected to continue to be considerably greater than the
supply, a fair and equitable organ sharing system is critical to the
future of a national transplant program that the public will support.''
S. Rep. No. 101-530 at 7 (1990) (The 1990 amendments also required that
the OPTN report on comparative costs and patient outcomes at all
transplant centers). As discussed in more detail below, in 1986 the
Congress also amended the Social Security Act to make OPTN membership,
and compliance with allocation policies approved by the Secretary,
mandatory rather than voluntary for Medicare-participating hospitals
and all organ procurement organizations.
Thus, the Congress envisioned an equitable national system that
would be
[[Page 16298]]
operated by the transplant community--including physicians and
officials of transplant facilities as well as other specialists and
individuals representing transplant patients, their families, and the
general public--with oversight by HHS.
Human organs that are donated for transplantation are a public
trust. These regulations are intended to ensure that donated organs are
equitably allocated among all patients, with priority to those most in
need in accordance with sound medical judgment. These regulations also
complement the recently announced National Organ and Tissue Donation
Initiative. The initiative addresses the fact that organ donation has
not kept pace with the need. Only about a third of potential cadaveric
donations are made; and, when families are asked, only about half give
consent. The initiative seeks to improve the number of potential donors
identified and asked to donate organs. This improvement would be
accomplished through proposed rules, published in the Federal Register
on December 19, 1997, which would require Medicare-participating
hospitals to work more closely with local organ procurement
organizations. A similar approach was adopted by the Commonwealth of
Pennsylvania, effective March 1995. By 1997, a 40 percent increase in
organ donors and a 49 percent increase in organ transplants had taken
place in southeastern Pennsylvania.
The initiative also seeks to improve the percentage of donations
when requests are made to donate. The initiative will accomplish this
goal by working with a number of partners to eliminate barriers to
donation, such as the failure of individuals wishing to donate organs
to discuss their wishes with their families. The initiative also seeks
to learn more about what works to increase organ donation and to
disseminate that knowledge broadly.
Advances in medical science and technology have made organ
transplantation an increasingly successful and common medical
procedure. Experience performing transplants and the development of
better immunosuppressive regimens have increased the survival rates for
transplant recipients. Comparing data for transplants performed in 1988
with data for transplants performed in 1995, one year patient survival
rates increased as follows: livers, from 81 percent to 87 percent;
hearts, from 83 percent to 85 percent; and lungs from 50 percent to 77
percent.
In addition, technological advances have made broader geographic
sharing possible. For example, the use of the Belzer UW solution,
developed in the 1980s, has dramatically increased both graft survival
rates and the time in which the organ survives out of the body. This
``cold ischemic time'' is used to transport an organ to a potential
recipient.
This rule is intended to ensure that organ allocation policies are
continuously reevaluated and revised to meet the statutory goal of
equitable national allocation of organs in accordance with medical
criteria.
This rule provides the framework for OPTN activity by clarifying
how the essential functions of the OPTN should be conducted in order to
better achieve an equitable national system.
Several evaluations of organ allocation have recommended a truly
national waiting system for organ allocation. A 1990 evaluation of the
OPTN conducted by Abt Associates recommended that the OPTN develop a
national patient-focused system:
Unless there is a clear disadvantage to patients or procurement
in having a single national list for each organ, the OPTN should
move towards a single national list and develop point schemes that
minimize cold ischemic and transplant times.
Evaluation of the Organ Procurement and Transplantation Network, at 85
(Abt Associates, August 21, 1990)
The HHS Office of Inspector General reached similar conclusions,
finding that ``current organ distribution practices fall short of
congressional and professional expectations,'' and that ``[t]here has
been substantial progress in developing a national organ distribution
system grounded in uniform policies and standards. However, organ
distribution remains * * * confined primarily within the individual
service areas of the * * * Organ Procurement Organizations.'' The
Distribution of Organs for Transplantation: Expectations and Practices
at 8, 13 (Office of Inspector General, March 1991).
Current OPTN organ allocations policies still do not create the
truly national system intended by the statute. Current OPTN allocation
policies do not reflect the more equitable, broader sharing possible
under current views of appropriate cold ischemic time. These policies
nominally give priority to the life or death needs of the sickest
patients, but the resulting allocation schemes fall short of that
objective. By allocating organs primarily at the local level, OPTN
policies give the sickest patients a substantially lower chance at
being promptly matched to a suitable organ (and thereby receiving a
potentially life-saving transplant) than would be the case with broader
geographic sharing.
At the national level, these policies treat patients inequitably
because they create enormous geographic disparities in the time
patients must wait to receive transplants. This approach is
inconsistent with the views of transplant candidates and the general
public who, according to a 1994 OPTN-initiated survey, were likely to
give top priority to the policy that ``makes waiting time about the
same for all patients nationally.'' Page 8 of the United Network for
Organ Sharing (UNOS) comments on the NPRM, December 6, 1994. In effect,
these policies treat the sickest patients differently depending on
where they live or which transplant hospital's waiting list they are
on. This result also is inconsistent with the views of at least half of
transplant recipients and candidates, who, according to the same
survey, ``would give top priority to a patient who is the most
critically ill and has the least time to live.'' Page 7 of UNOS
comments. Finally, this approach is inconsistent with the views of a
blue ribbon panel that examined a broad range of issues pertaining to
organ transplantation, including the technical, practical, and ethical
limitations on sharing organs. The panel noted:
The principle that donated cadaveric organs are a national
resource implies that,
In principle, and to the extent technically and practically
achievable, any citizen or resident of the United States in need of
a transplant should be considered as a potential recipient of each
retrieved organ on a basis equal to that of a patient who lives in
the area where the organs or tissues are retrieved. Organs and
tissues ought to be distributed on the basis of objective priority
criteria, and not on the basis of accidents of geography.
Report of the Task Force on Organ Transplantation, April 1986 at 91
(quoting Hunsicker, LG)
Another flaw in current OPTN policies pertains to disclosure of
information. The statute requires the Secretary to provide information
to patients, their families, and physicians about transplantation.
Current policies in this area do not give patients, their families, and
physicians the timely information they need to help in selecting a
transplant hospital. For example, one-year survival rates of patients
and organ grafts are valuable information in comparing the relative
effectiveness of transplant programs. However, today a patient seeking
this information would have to rely on four year old OPTN data released
in 1997. Moreover, these data are contained in
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nine volumes with 3,200 pages. A patient seeking to compare centers
would find these data difficult to use. In addition, access to
accurate, timely data will enable the Department to monitor the
effectiveness of organ transplantation and provide the general public
with information on how well the transplantation network is performing.
The National Organ Transplant Act vested in the Secretary oversight
of the OPTN and responsibility for ensuring public benefit. Amendments
to the Social Security Act in 1986 underscored the Secretary's role.
Working in partnership with the transplant community, the Secretary has
final authority over OPTN policies and procedures. In particular, the
Secretary has a statutory mandate not only to ensure that the OPTN
distributes organs ``equitably'' and fulfills other statutory
requirements but also to obtain and act upon ``critical comments
relating to the manner in which (the OPTN) is carrying out the duties
of the Network.'' The Secretary has chosen to issue regulations for the
purpose of ensuring that the system evolves to keep pace with
improvements in technology and medical science (such as improvements in
organ preservation technology and reductions in the disparities in
survival rates among more sick and less sick patients) and is operating
effectively and efficiently to meet its statutory goals.
Six principles underlie this regulation:
Transplant patients are best served by an organ allocation
system that functions equitably on a nationwide basis;
The Secretary of Health and Human Services should
represent the public interest by setting broad goals for the OPTN and
by overseeing OPTN policy development and operations with a view toward
ensuring that the goals are being addressed in a reasonable manner;
The OPTN must exercise leadership in performing its
responsibilities under the National Organ Transplant Act, in particular
by devising the specific policies assigned under these regulations, and
by adapting its policies and procedures to changes in medical science
and technology;
Organs should be equitably allocated to all patients,
giving priority to those patients in most urgent medical need of
transplantation, in accordance with sound medical judgment;
Thorough, timely, and easy to use information about
transplant centers, including center-specific performance data, is
essential for measuring quality of care and should be readily available
to help patients and physicians in choosing among transplant centers;
Potential conflicts of interest should be minimized for
those who are responsible for operation of the OPTN.
B. Legislative and Regulatory History
The OPTN was established under section 372 of the PHS Act, as
enacted by the National Organ Transplant Act of 1984 (Pub. L. 98-507),
and amended by Pub. L. 100-607 and Pub. L. 101-616. Section 372
requires the Secretary to provide by contract for the establishment and
operation of the OPTN to manage the organ allocation system, to
increase the supply of donated organs, and to perform related and other
activities.
Until the enactment of the Omnibus Budget Reconciliation Act of
1986 (Pub. L. 99-509), membership in the OPTN was voluntary. Section
9318 of Public Law 99-509 added a new section 1138 to the Social
Security Act. Section 1138(a)(1)(B) requires hospitals that perform
organ transplants to be members of and abide by the rules and
requirements of the OPTN as a condition for participation in the
Medicare and Medicaid programs. This requirement places at risk the
transplant hospitals' participation in these programs, not just
payments for transplantation, and as a practical matter makes the
hospitals' survival dependent on following such rules and requirements.
Section 1138(b)(1)(D) requires that to be eligible for reimbursement of
organ procurement costs by Medicare or Medicaid an OPO must be a member
of and abide by the rules and requirements of the OPTN.
Section 102(c) of the Balanced Budget and Emergency Deficit Control
and Reaffirmation Act of 1987 (Pub. L. 100-119) delayed the effective
date of Sec. 1138(a) of the Social Security Act concerning hospitals
from October 1, 1987, to November 21, 1987, and Sec. 4009(g) of the
Omnibus Budget Reconciliation Act of 1987 (Pub. L. 100-203) further
delayed the effective date of Sec. 1138(b) of the Act concerning OPOs
to April 1, 1988.
The Organ Transplant Amendments of 1988 (Title IV of Pub. L. 100-
607) amended Sec. 372 of the Public Health Service Act to require that
the OPTN establish membership criteria and subject its policies to
public review and comment.
On March 1, 1988 (53 FR 6526), the Department published final rules
that included the requirement that Medicare/Medicaid participating
hospitals that perform transplants, and designated OPOs, be members of
and abide by the rules and requirements of the OPTN (42 CFR 485.305
(now 42 CFR 486.308) and 482.12(c)(5)(ii)) in order to qualify for
Medicare or Medicaid payments.
On December 18, 1989, the Department published a Federal Register
Notice (54 FR 51802) addressing the oversight of the OPTN. In that
Notice, the Secretary stated that no OPTN policies would become legally
binding ``rules or requirements'' of the OPTN for purposes of section
1138 until or unless they were approved by the Secretary.
The 1994 proposed regulations (59 FR 46482) were intended to
implement that decision, as is this final rule with comment period. In
those proposed regulations, the Secretary raised a wide range of
issues, including procedures for joining the OPTN, the Federal review
processes, procedures and standards for information collection and
dissemination; membership requirements and compliance procedures; and
the criteria for allocation of each of the solid organs. On November
13, 1996, the Secretary issued a Federal Register notice reopening the
comment period and announcing a public hearing to be held in December
1996, to address issues raised by those proposed regulations, and to
hear ideas regarding increasing organ donation and the controversial
and difficult problems surrounding organ allocation generally and liver
allocation policies in particular. From December 10 to 12, 1996, that
hearing was held. As under the proposed regulations, the final rule
provides for Federal oversight of the processes by which the OPTN
allocates organs for transplantation. It focuses the Federal role on
ensuring that those processes and resulting policies are equitable,
provides for broader public participation and Secretarial review, and
includes access to information for patients and their families and
physicians.
Under the final regulations, the OPTN has responsibility for
developing medical criteria for patient listing, medical urgency
criteria (``status'' definitions), organ allocation policies, other
policies governing organ transplantation, and policies for the day-to-
day operation of the OPTN. The Secretary has responsibility for
oversight of the OPTN, for establishing performance goals and
indicators to guide the national system for distribution of organs, and
for final approval of those OPTN policies that are to be enforceable.
Both the OPTN and the Secretary have responsibility for dissemination
of information to the
[[Page 16300]]
public, including patients, physicians, payers, and researchers.
This final rule was developed after consideration of comments from
all elements of the transplant community on the entire range of issues.
Comments were received not only during the original comment period but
also during the last two years and attendant to the public hearing held
in December 1996. Although the Secretary believes that this rule
addresses all of the major issues and questions that had been
identified, the Department remains open to suggestions for further
improvements. The Department has provided for additional public
comments on these regulations to be submitted during the next 60 days.
The Department will also provide for public input on OPTN proposals for
policies to implement these regulations.
C. DHHS and OPTN Relationships
The public comments indicate that many persons misunderstand the
role of the OPTN. The OPTN is sometimes characterized as a voluntary
system through which consensus decisions are reached as to how to
allocate organs among patients (who may live or die based on these
decisions). The underlying statutes, absent Secretarial oversight, give
the OPTN authority from which individual patients, physicians, and
hospitals have little recourse. If the OPTN changes organ allocation
criteria, it may advantage some patients and disadvantage others
because there are not enough organs donated to meet the need and no
alternative organ allocation entity exists. The unique role of the OPTN
thus gives rise to a fundamental question. To what process or remedy
can patients, their families, physicians, or members of the general
public turn if they wish to question policies, decisions, procedures,
or practices of the OPTN? By providing a framework for OPTN policy
development and describing the role of the Secretary therein, this rule
addresses that question.
The United Network for Organ Sharing (UNOS), a private corporation,
operates the OPTN under contract with the Department. The contract is
subject to the competitive bidding process. Under recent Requests for
Proposals, there have been no effective competitors to the current
contractor. The current contract expires September 30, 1999.
As a private organization, UNOS has by-laws, operating procedures,
and membership requirements. They apply only to UNOS members and not to
OPTN members. Membership in UNOS is not a requirement for membership in
the OPTN. Therefore, such procedures are not OPTN procedures, and
because they do not bind OPTN members, they are not the subject of this
regulation. Because OPTN members are not required to become UNOS
members, UNOS procedures are subject to these regulations only if they
conflict with OPTN requirements, or if they conflict with the terms of
the contract for the operation of the OPTN, or these regulations. For
example, UNOS may impose conditions for membership in UNOS, but those
conditions may not be substituted for, or used to augment, the
regulatory requirements for the UNOS-administered OPTN. In contrast,
matters relating to the OPTN are encompassed by these regulations; and
UNOS, as the OPTN contractor, is required to comply with these
regulations and to issue policies consistent with the requirements of
these regulations.
The Department believes that the transplantation network must be
operated by professionals in the transplant community, and that both
allocation and other policies of the OPTN should be developed by
transplant professionals, in an open environment that includes the
public, particularly transplant patients and donor families. It is not
the desire or intention of the Department to interfere in the practice
of medicine. This rule does not alter the role of the OPTN to use its
judgment regarding appropriate medical criteria for organ allocation
nor is it intended to circumscribe the discretion afforded to doctors
who must make the difficult judgments that affect individual patients.
At the same time, the Department has an important and constructive role
to play, particularly on behalf of patients. Human organs that are
given to save lives are a public resource and a public trust.
The process adopted in this rule strikes a balance among these
important principles. When the OPTN develops policies, or when
complaints are raised concerning OPTN policies, the regulation allows a
number of options. The Secretary may approve an OPTN-proposed policy or
find that the complaint has no merit. The Secretary also may take
another approach depending on the issues presented. For example, the
Secretary: may seek broader public input on the issue; may determine
whether violations of OPTN-proposed policies should carry any one of a
range of consequences--no consequence, loss of membership in the OPTN,
or loss of a hospital's ability to participate in Medicare and
Medicaid; may provide comments for the OPTN's consideration; may direct
the OPTN to adopt a policy; or, may develop a policy that the OPTN must
follow. An example of this last option is this regulation's provisions
prescribing who the OPTN must admit as members. Instead of an
exhaustive listing of these and other options, the regulation, at
sections 121.4 (b)(2) and (d) simply provides that the Secretary may
``take such other action as the Secretary determines appropriate.''
Questions have also arisen about the relationship of OPTN policies
to other standards and requirements. A number of Federal statutes,
including those relating to Medicare and Medicaid, civil rights, fraud
and abuse, clinical laboratories, organ procurement, control of
infectious disease, and regulation of blood and blood products, have
provisions that may affect, or be affected by, the policies of the
OPTN. For example, several years ago the Department made decisions as
to the required qualifications for clinical laboratory directors, after
an extended public comment process. Those decisions did not impose the
most stringent possible academic qualifications because the available
evidence did not show that those levels were necessary for high
performance. Any OPTN policy that directly or indirectly would require
member hospitals to do business only with laboratories with directors
meeting a higher qualification would conflict with the HHS regulation,
and thus not be binding upon OPTN members unless the Secretary approved
that policy as an OPTN requirement.
In order to prevent such problems, this regulation creates a system
in which the OPTN has three options whenever it identifies a policy
that it believes will contribute to high performance: the OPTN can
recommend its use by members; the OPTN can request that HHS make it
enforceable, or the OPTN can petition HHS to modify other regulations
(such as clinical laboratory or blood regulations) to adopt that
policy. What the OPTN cannot do is unilaterally impose a policy that
has the effect of, or changes the terms of, a national policy already
subject to the oversight of a cognizant Federal agency.
The Secretary will review the OPTN policies that may interact with
other statutes or with rules promulgated through other Federal
programs. To clarify the policy development and review process, we have
added a new Sec. 121.4, Policies: Secretarial Review, and Appeals,
which consolidates regulatory requirements from proposed Secs. 121.3,
121.7, and 121.10. The addition of new Sec. 121.4 results in
renumbering Secs. 121.4-121.12. See the discussion at section II(C6),
under Supplementary Information, below.
[[Page 16301]]
D. Enforcement
Some of the comments received in response to the Notice of Proposed
Rulemaking or delivered at the public hearings indicate that there may
be misunderstandings about the relationship between section 1138 of the
Social Security Act and the OPTN regulations, and their respective
enforcement provisions.
1. Section 1138 of the Social Security Act
As discussed above, section 1138 requires Medicare and Medicaid
participating hospitals that perform transplants to be members of the
OPTN and abide by its rules and requirements. Section 1138 also
contains similar requirements for OPOs in order for organ procurement
costs attributable to payments to an OPO to be paid by Medicare and
Medicaid. These requirements are also found in final rules (42 CFR
485.305 (now 42 CFR 486.308) and 482.12(c)(5)(ii)) published on March
1, 1988 (53 FR 6526). Further, on December 18, 1989, the Department
published a general notice in the Federal Register (54 FR 51802)
announcing that, in order to be a rule or requirement of the OPTN and
therefore mandatory or binding on OPOs and hospitals participating in
Medicare or Medicaid, the Secretary must have given formal approval to
the rule or requirement. Violations of section 1138 could result in
withholding of reimbursement under Medicare or Medicaid.
Section 1138 and the final rules and general notice that followed
pertain only to OPOs and hospitals participating in Medicare or
Medicaid. In its general notice, the Department intended to define what
is meant by a ``rule or requirement of the OPTN'' for the purposes of
implementing section 1138. In applying the policy in the general
notice, the Department considers a ``rule or requirement of the OPTN''
to be those rules developed as provided for in these regulations.
Two examples illustrate the significance of this provision. First,
an OPO or transplant hospital participating in Medicare or Medicaid
could be considered in violation of section 1138 if the Secretary found
that it did not provide information to the OPTN as required
specifically by Sec. 121.11(b)(2) or that it procured for
transplantation organs known to be infected with the human
immunodeficiency virus, prohibited specifically by Sec. 121.6(b).
Conversely, these institutions would not be considered in violation of
section 1138 if they were found by the Secretary to be acting contrary
to a policy implemented by the OPTN but not formally approved by the
Secretary as enforceable. Second, if an OPTN member procured and
arranged for allocation of donor kidneys in a manner inconsistent with
the OPTN's kidney allocation policy as in effect in 1996, it would not
be considered in violation of section 1138, because that allocation
policy is not approved by the Secretary as enforceable policy.
Therefore, policies of the OPTN that are not articulated in these or
subsequent OPTN regulations or elsewhere approved by the Secretary are
not enforceable under Sec. 121.10.
2. OPTN Policies
There has been discussion about whether all OPTN policies should be
enforceable. The Secretary believes that compliance with existing
voluntary policies has been excellent. Furthermore, some commenters at
the public hearings expressed support for the current role of the OPTN
in devising and issuing such policies. Finally, the field of organ
transplantation is dynamic, yielding technological advances that the
OPTN must accommodate as quickly as possible if patients are to receive
their full benefits. It can do so efficiently under this tested
approach. Therefore, the Secretary has decided to continue this
approach.
The Secretary recognizes, however, that compliance with certain
policies, such as those relating to organ allocation, are crucial to
the success of the OPTN and expects the OPTN to monitor compliance with
these policies closely under Sec. 121.10. If violations are widespread,
or if uniform compliance is essential, the Secretary will consider
making such policies enforceable. The Secretary also recognizes the
need for additional public participation in the development of some
OPTN policies, such as fundamental revisions to organ allocation
policies, and has included in this rule provisions that (1) require the
OPTN Board to provide opportunity for the OPTN membership and other
interested parties to comment on all of its proposed policies, (2)
enable the Secretary to seek comment from the public and to direct the
OPTN to revise policies if necessary, and (3) provide timely access to
information for patients, the public, and payers. These provisions are
discussed further in section II.
The requirements that are explicit in this final rule are subject
to its enforcement provisions. For example, if a transplant program did
not establish organ acceptance criteria and provide such criteria to
the OPOs with which they are affiliated and to the OPTN, as required
specifically by Sec. 121.6(c), it could be found to be out of
compliance with the OPTN regulations and subject to suspension of its
designated status under Sec. 121.9, as discussed further in section II.
II. Summary of Public Comments and Policies of the Final Rule
In addition to public comments directed specifically to the NPRM
document, the Department has received other comments and
recommendations directed at issues covered by this final rule, as well
as additional documents described below. Much of this additional
information was received during 1996 and 1997, subsequent to the
original rulemaking dates. In particular, the Secretary determined in
1996 that there were sufficient controversies to justify reopening the
comment period and scheduled a three-day public hearing, subsequently
held on December 10-12, 1996.
The information received since the close of the original comment
period falls into several broad categories. First, the OPTN itself has
considered or adopted a substantial number of policy changes, each
accompanied by supporting information presented to the OPTN Board of
Directors and to the public. Second, the transplant community,
including the OPTN, has created additional materials. Both the OPTN and
the University of Pittsburgh sponsored the development of simulation
modeling to estimate the likely effects of alternative liver allocation
policies (the ``Pritsker'' and ``CONSAD'' models discussed later in
this preamble). Third, approximately 110 persons individually or
representing the OPTN, patients and patient organizations, transplant
institutions, and professional associations testified at the December
1996 public hearing; and hundreds of others sent written comments.
Finally, the Secretary considered other materials including, for
example, correspondence from Members of Congress and a number of recent
newspaper articles which focused on organ transplantation issues and
controversies.
The testimony and comments received in connection with the public
hearing contain a total of 541 documents, with 667 signatures. Of
these, 180 signatories are identifiable as transplant recipients or
candidates or their families and friends, 327 as physicians, and 43 as
other health personnel such as nurses, hospital administrators, and
directors of organ procurement organizations. National organizations
submitted 30 documents.
[[Page 16302]]
Twenty-two petition letters contain a total of 5,462 signatures. No
attempt has been made to identify the signatories of the petition by
type.
Among the documents in the docket room at 12420 Parklawn Drive,
Room 123, Rockville, MD and available for review or copying are the
actual comments as well as a summary and analysis of all of the
comments received in response to the NPRM and the December 1996 public
hearing, the 1996 Annual Report of the OPTN and Scientific Registry,
the 1996 Code of Medical Ethics of the Council on Ethical and Judicial
Affairs of the American Medical Association, the 1993 white paper ``The
Principles of Equitable Organ Allocation'' of the OPTN Ad Hoc Committee
on Organ Allocation, the materials prepared for the OPTN Board of
Directors before each Board Meeting over the last several years, the
1991 report of the HHS Inspector General entitled ``The Distribution of
Organs for Transplantation: Expectations and Practices,'' the 1993
report of the General Accounting Office entitled ``Organ Transplants:
Increased Effort Needed to Boost Supply and Ensure Equitable
Distribution of Organs,'' the OPTN's multi-volume ``Report of Center
Specific Graft and Patient Survival Rates'' for both 1994 and 1997, a
1995 report from the CONSAD Research Corporation providing ``An
Analysis of Alternative National Policies for Allocating Donor Livers
for Transplantation,'' a number of computer simulations on liver
allocation policy prepared by the Pritsker Corporation in 1996 and 1997
(most included in the OPTN Board materials listed above), a number of
computer simulations on liver allocation policy prepared by CONSAD in
1996 and 1997, a series of investigative articles on organ
transplantation and allocation issues that appeared in the Cleveland
Plain Dealer in early 1997, other newspaper articles, and a GAO report,
``Organ Procurement Organizations, Alternatives Being Developed to More
Accurately Assess Performance'', published in November, 1997.
In addition, this rule and some of the documents listed above--such
as the transcript of the public hearings--are available on the HRSA Web
site at http://www.hrsa.dhhs.gov/bhrd/dot/dotmain.htm.
A. Summary of Original Public Comments
The preamble to the Notice of Proposed Rulemaking (NPRM) asked the
public to comment separately on the specific provisions of the proposed
rule and on the individual policies then in effect voluntarily under
which organs were being allocated to potential transplant recipients.
Of the 121 letters received, 59 contained comments on specific sections
of the NPRM, 60 on the allocation policies, and two commented on both.
About half of the original comments are addressed in the discussion of
public comments on allocation policies, below.
All but two of the 61 letters commenting on specific sections of
the NPRM other than allocation policy were from individuals identified
with organizations. National groups included the Ad Hoc Coalition on
Organ Transplantation, the American Association of Kidney Patients, the
American Center for Transplant Resources, the American Society of
Histocompatibility and Immunogenetics, the American Society of
Transplant Physicians, the American Society of Transplant Surgeons, the
Association of Organ Procurement Organizations, the National Kidney
Foundation, the North American Transplant Coordinators Organization,
and the United Network for Organ Sharing. Thirty-two letters were from
individuals affiliated with hospitals, ten from organ procurement
organizations, one from a law firm representing a hospital, two from
members of the U.S. House of Representatives, one from a former member
of Congress, and two from individuals who identified themselves as
organ transplant recipients.
The 61 letters presented a total of 210 comments on specific
sections of the NPRM as follows: Sec. 121.2--Definitions (17);
Sec. 121.3--Composition of the OPTN (41); Sec. 121.4--Listing
Requirements (18); Sec. 121.5--Organ Procurement (6); Sec. 121.6--
Identification of Organ Recipient (24); Sec. 121.7--Allocation of
Organs (40); Sec. 121.8--Designated Transplant Program Requirements
(34); Sec. 121.9--Review and Evaluation (2); Sec. 121.10--Appeals of
OPTN Policies and Procedures (2); Sec. 121.11--Record Maintenance and
Reporting Requirements (26). These comments are discussed below in the
context of those specific sections.
B. Summary of Public Hearing
The public hearings demonstrated that there is considerable
controversy over many aspects of organ allocation policy, along with
many areas of agreement. A number of fundamental questions were
addressed by multiple witnesses, and their comments on these and the
Secretary's decisions are summarized below. The Department's Federal
Register Notice establishing the agenda for the hearing focused on two
issues: Increasing organ donation and liver allocation policy--but
those who testified raised many additional issues.
1. What Role Should the Federal Government Have in Organ Allocation
Policy?
Partly as a result of the controversy surrounding the new OPTN
liver allocation policies proposed in 1996, some individuals questioned
whether the private sector can or should set policy for a system that
has such a profound effect on life and death decisions. The recurring
view expressed in testimony, however, was to preserve the current
contractual arrangements for the operation of the OPTN, but for HHS to
exercise closer oversight, particularly in organ allocation policy.
Others testified to the contrary, arguing that the OPTN was dominated
by the self-interest of transplant physicians and surgeons (see
discussion below) and that only the government could take an impartial
role in a field so dominated by conflicting interests.
Despite support for the OPTN contract and the structure of the
OPTN, a number of individuals and organizations argued that the
approval of a flawed liver allocation policy in November 1996 (see
below), and the failure to improve current policy in more fundamental
ways illustrated systemic flaws in the current governance structure.
One line of comments focused upon the structure of the OPTN Board of
Directors, which was characterized (incorrectly) as giving each
transplant hospital one vote, without regard to the number of patients
on the waiting list or the number of individuals transplanted. Some
patients, patient groups, and directors of the larger programs
advocated models where patients' interests would have greater
representation. Others argued that the OPTN is dominated by hospitals--
large and small--and transplant surgeons and physicians and that the
larger public interest, the altruistic interests of donors and donor
families, and interests of potential recipients are neglected.
As discussed elsewhere in this preamble, the Secretary believes
that the Department has an important and constructive role to play,
particularly on behalf of patients.
2. Are the Liver Allocation Policies That the OPTN Adopted in November
1996 Fair?
The OPTN Board had approved a new liver allocation policy shortly
before the public hearing. At the public hearing and in the comments
received, many patients with chronic liver disease
[[Page 16303]]
opposed the new policy; most physicians supported it. Table 1 presents
the pertinent data.
Table 1.--Opinions by Type of Respondent (Excluding Petitions)
------------------------------------------------------------------------
Pro new Con new
Category policy policy
------------------------------------------------------------------------
Physicians........................................ 136 5
Other health personnel............................ 13 3
Recipients/candidates and families................ 31 128
---------------------
Totals........................................ 180 136
------------------------------------------------------------------------
Patients and their advocates asserted that their chance to receive
an organ had been decreased significantly by the new policy of
transplanting patients with acute hepatic failure and primary non-
function before chronic patients who were also in intensive care units
and had equally short life expectancies. Moreover, patients and their
advocates asserted that there was no significant medical argument
favoring preference for the ``acute'' group. (OPTN data tend to confirm
this assertion and show that the acute patients do not have an
appreciably better post-transplant survival rate than the chronic
patients, as discussed later in this preamble). They pointed out that,
despite the prospect of imminent death, they were newly downgraded into
a lower priority group of patients and that all chronic patients were
being grouped together rather than differentiating among chronic
patients and their varying medical conditions. Strong pleas were made
by some medical personnel, patients, and patient advocacy groups for a
system of classification based on objective and relevant medical
criteria and for broader sharing of organs.
Most OPTN officials defended the new policies but based these
arguments on the extensive and prolonged committee processes involved
rather than medical data. However, the Chairman of the OPTN Patient
Affairs Committee indicated that the needs of the chronic disease
patients had not been considered carefully enough when the new policy
was evaluated by his committee. He stated that the OPTN, while
attempting to accomplish good purpose for one group of patients, had
apparently disadvantaged another group with equally high medical
urgency. He also promised to have his committee reconsider its
position.
Some commenters urged that a moratorium be placed on the
implementation of the new policy until the needs of the chronic
patients could be properly considered. As a result of the airing of
these issues at the hearing, the OPTN established this moratorium
shortly after the hearing. In further response, in June 1997, the Board
of Directors voted to implement a new policy that would reform the
controversial policy to some degree. The newer policy places very sick
chronic patients in a separate status subgroup and also assigns them a
second priority--i.e., after the acute patients. However, as explained
in greater detail below, it reduces, but does not eliminate, the
disadvantage that had been imposed on chronic patients in 1996.
This rule requires the OPTN to promptly take a fresh look at its
current policies in light of the rule's performance goals.
3. Should Transplantation Be Centralized in a Few Centers That Meet
More Stringent Criteria, or Are There Advantages to the Present
Geographic Distribution of Programs?
Although the Department had not identified establishing volume or
performance criteria for individual hospitals as a hearing topic, some
commenters raised this issue. This issue arises because, although
patients are free (subject to insurance coverage) to select from among
most transplant hospitals in the United States, under current OPTN
policies, the number of organs available to a hospital in a particular
area does not rise or fall as the number of patients increases or
decreases but is largely dependent on the number of donors in that
local area. As a consequence of a ``local first'' allocation policy,
most organs leave the local area only if there are no local patients
who could use the organ. (An exception is ``no mismatch'' kidneys,
which are shared nationally.) As a result of hospitals drawing
primarily from the local pool of donated organs, no hospital can expand
its program beyond the local supply of organs without disadvantaging
the patients who choose it. Representatives of some small-volume
transplant programs argued that broader geographic sharing might result
in local, smaller hospitals being forced to close their transplant
programs.
The argument for wider sharing of organs was made vigorously by
representatives of some large-volume transplant programs. They also
argued that the quality of performance and outcome was related to the
number of procedures performed. The contrary argument--to recognize the
importance of the small-volume programs--was made vigorously on the
basis of local and regional access to transplants and with testimony
and data suggesting that many small programs have outcomes equal to or
better than the larger hospitals. In addition, some patients expressed
concern about losing their system of support (family and neighbors) if
they had to leave their homes or communities to receive a transplant.
Another concern was the extra expense incurred by patients having to
move outside the home community for a transplant.
After the hearing, the Department determined, however, that this
concern over local access and increased travel only affects a small
number of patients. About half of liver patients must travel outside
their local area to obtain a transplant simply because almost all rural
areas, most cities, and about a dozen States have no liver transplant
programs. Also, the great majority of small-volume programs are located
in the same metropolitan area as large-volume programs. Thus, very few
patients might have to face this potential problem.
Some argued that the more remote the large hospital may be from a
needy patient, the greater the travel costs and the more likely those
without insurance or those with lower income will be effectively
excluded from the opportunity to receive an organ. On the other side,
some argued that larger programs have been more willing to list the
sicker patients and those with less ability to pay. The Department
finds these arguments speculative. About half of all patients have to
travel anyway, and nothing other than anecdotal evidence was presented
regarding how many patients are taken as charity cases at hospitals,
large or small.
It was argued that the Health Care Financing Administration and
some other large payers such as managed care organizations refer their
patients to higher volume programs and, thus, strain a system already
under stress because of the shortage of organs. Others argued that the
organ shortage is the same regardless of where payers direct their
patients.
The Secretary concludes that there is no persuasive evidence that
the provisions of this rule--equitable sharing of organs, based on
objective criteria of medical urgency and free patient choice among
transplant programs--will damage transplant institutions of any size.
However, in this regard, the Department also will consider whether any
demonstrable institutional impact will result from the policies to be
developed by the OPTN.
[[Page 16304]]
4. Should Organs Be Shared Across Geographic Lines--Regionally or
Nationally?
Many patients and patient advocates, and some hospital
representatives, argued that organs should ``follow'' the patient. That
is, regardless of where a patient lives or lists, he or she should have
the same chance of receiving an organ as if living or listing
elsewhere. Local preference prevents this result, and proponents of
this view opposed local preference. Why should some patients who list
in areas that, for whatever reason, obtain more organs in relation to
local demand benefit over patients from other areas who have equal or
greater medical need? Why should other patients in those same areas who
are sicker nevertheless not receive a matching organ from another area?
Another argument against local preference is that it limits the ability
of patients to select the medical program and physician they prefer.
The patients of large payers are also disadvantaged if organs are not
allocated where the patient will get her or his care, unless the payer
is willing to make special arrangements to move patients where waiting
lists are shortest or to ``multiple list'' patients at more than one
transplant hospital because of long local waiting times. Patients or
payers who consider ``multiple listing'' are also, in effect, forced to
choose between using local providers and, potentially, cross-
continental travel simply to have a good chance of getting a organ.
Some argued that the feasibility of national organ sharing is
limited by the cold ischemic time (the time after procurement that an
organ remains viable for successful transplantation). Witnesses said
that this time ranges from 12 to 18 hours for livers and that, for
livers transplanted in less than this time, there is little difference
in graft survival attributed to cold ischemic time. (Compared to
livers, the cold ischemic time is much shorter for hearts and much
longer for kidneys.) Some commenters argued that travel times to and
from large cities, where most transplant hospitals are located, readily
permits a national allocation scheme for livers. However, others
argued, travel times from small communities (the locale of many donors)
to large cities or to other small communities are not always
predictable and that estimates of travel time are not always reliable.
Proponents of national sharing of livers pointed out that other
organs--including hearts and kidneys--are successfully shared outside
of the local area and that many livers were nationally shared for the
sickest patients until 1991. These witnesses argued that the
transportation argument was irrelevant since any sensible policy would
be designed to ensure that organs would not be transported in cases
where this would result in waste.
Some witnesses argued that sharing of organs across geographic
lines would just ``switch the zip codes'' of those who died. This
reflects the stark reality that, so long as the number of organs is
insufficient to transplant all those in need, some persons are likely
to die while awaiting a transplant. Proponents of broader sharing
countered that the OPTN's own modeling showed that lives could be saved
if organs went to the sickest patients first within broad geographic
areas rather than giving preference to local patients who, though ill,
were not in imminent danger of death.
Among the arguments made against broader sharing was that this
could harm local procurement. Those taking this view emphasized the
value of the relationships between the transplant hospitals and their
local organ procurement organization and asserted that local allocation
tends to promote organ donation and retrieval by local transplant
surgeons. A related argument was advanced against broader sharing
suggesting that, if referring physicians perceive organs are always
``shipped out'', they will be dissuaded from referring donors. However,
those in favor of broader sharing argued that there was no evidence to
support the local preference argument. They stated that donor families
have no preference where the organ is used, believing that donor
families want only that their loved one's organs help individuals most
in need.
In this regard, a 1994 OPTN survey (reported in the UNOS Update of
July 1994) shows that the overwhelming majority of donor families state
as their preference that organs go to the neediest patients, regardless
of geography, so long as organs are not wasted. That same survey showed
very high support for equalizing waiting times. Many commenters noted
that, even under the current system of local priority, some organs are
shared regionally or nationally. HHS has seen no credible evidence that
local preference encourages donation or that sharing organs regionally
or nationally for the sickest patients will impact organ donation. Nor
is there any evidence that transplant professionals perform differently
when the retrieval is for a distant patient rather than a local
patient.
5. Which Is Preferred, Transplanting the Sickest First or Transplanting
Patients Who Are Most Likely To Survive the Greatest Number of Years?
Many witnesses at the public hearing agreed on two broad points:
first, from the perspective of an individual patient who is at risk of
imminent death, the ``sickest first'' policy is the only choice; and
second, there are patients who are so likely to die that it would be
futile to transplant them and waste an organ that could have saved
someone else. Some argued that transplantation before a patient becomes
``sickest'' provides better outcomes and longer graft and patient
survival, and increases the supply of organs by reducing the number of
second transplants. However, to adopt a policy favoring transplantation
of the least sick patients would mean that more hospitalized patients
might die. Moreover, the chronic liver patients asserted that their
expected survival rates were not only high, but also essentially equal
to those of acute patients, who were gaining preference. They
questioned how reducing their chance of living, when both urgency and
outcome were essentially equal, could meet any reasonable ethical
standard.
The available evidence shows that, for most patients, higher
medical urgency does not reduce the likelihood of post-transplant
survival to the extent that less ill patients should receive higher
priority. Although current OPTN policies vary by organ, the predominant
thrust of the OPTN policies is to give priority to greater medical
need. (These regulations are not intended to preclude considerations
underlying current allocation policies such as the judgment afforded
surgeons in individual cases, the needs of children and sensitized
patients, and the priority given to no antigen mismatches for kidney
patients.) The Secretary therefore concludes that ethical
considerations require that the most medically urgent patients--those
who are very ill but who, in the judgment of their physicians, have a
reasonable likelihood of post-transplant survival--receive preference
in organ allocation over those who are less medically urgent.
6. How Much ``Game Playing'' Exists in the Present System?
A number of witnesses asserted that the current system of organ
allocation and listing can be manipulated by hospitals, physicians, and
payers. Practices discussed included excluding high risk patients from
the list, listing patients early to gain waiting time points, listing
patients at more than one
[[Page 16305]]
transplant hospital to increase the chance of getting an organ, and
referring high risk patients to other hospitals to avoid adverse
performance outcomes. No data were presented in support of these
assertions, but they came from a cross-section of witnesses. Some
commented that the present debate evinces distrust among transplant
professionals--local hospitals work together and with the local OPO,
whereas non-local hospitals may be ``gaming'' the system to advantage
their patients. Presenters suggested modifications to the system to
minimize these tactics. Most supported the development of objective
medical criteria for listing and classifying candidates as a specific
reform that would increase fairness.
7. How Can HHS Promote and Facilitate an Increase in Organ Donation?
A plea for vigorous involvement of and leadership by HHS in organ
donation was almost unanimously supported. The diversity of experiences
and effectiveness among OPOs and hospitals, and variation among State
laws and practices, suggest a need for shared communication, education,
and Federal action. Many suggestions were offered to minimize
disincentives and maximize appropriate incentives for organ donation.
Emerging research data provide information about factors that influence
a donor family's decision to consent to offer a loved one's organs.
Many specific ideas were suggested for how government could invigorate
organ donation.
Toward that end, HHS is conducting a broad organ and tissue
donation initiative that implements many of the suggestions made at the
hearing, and others. Included as part of this initiative is a Notice of
Proposed Rulemaking published in the Federal Register on December 19,
1997 (62 FR 66725), which would require that hospitals refer all
appropriate deaths to OPOs, and that OPOs determine the criteria for
these mandatory referrals. In cooperation with other Federal agencies,
we are undertaking a major campaign to encourage Federal employees and
their families to volunteer to become potential organ donors. We also
encourage the transplant community to strengthen its various efforts to
increase organ and tissue donation, and to review whether transplant
hospitals are taking all reasonable steps to procure organs (a recent
review of OPTN data showed that about one-fourth of transplant
hospitals produced no donors in 1995). Finally, the Department will
host a conference to exchange information on identifying best practices
and promising innovations.
A number of surveys and studies have shown broad support for organ
donation generally. The Secretary believes the policies that are
contained in this rule will complement the initiative and build on this
public support for organ donation. Allocating organs nationally to
those most in need also will build on a broad base of public support.
As noted above, according to a 1994 OPTN-initiated survey, at least
half of transplant recipients and candidates ``would give top priority
to the patient who is the most critically ill and has the least time to
live.'' Page 7 of UNOS comments on NPRM, December 6, 1994. While some
commenters suggested that locally based allocation increases donation,
they did not offer any studies to support this suggestion. A 1991 HHS
Inspector General report rejects the notion of local use increasing
local donation. The Distribution of Organs for Transplantation:
Expectations and Practices at 15-16 (Office of Inspector General, March
1991). The same OPTN-initiated survey also discounts this approach,
concluding that ``Americans do not think that keeping an [donated]
organ in a specific locality is an important goal in and of itself. * *
* '' Page 8 of UNOS comments.
8. What Is the Responsibility To Provide Access to Transplantation
Services to All Americans, Regardless of Economic Status?
Access to transplantation services was described as being dependent
on a person's ability to pay, which virtually always requires health
insurance. A few State-supported hospitals testified that they accept
all patients regardless of ability to pay, but the preponderance of the
testimony was that most transplant hospitals require that the patient
demonstrate an ability to pay. As a result, commenters argued, the
promise to honor the altruistic gift of an organ to whoever needs it
most is being violated.
The Department cannot solve this problem under existing law or
through this rule. Nor are problems with the ability to pay unique to
transplantation. What is unique is the interest of the donor family in
fair allocation. The Secretary concludes that the Department and the
OPTN should give more emphasis to socio-economic equity in
transplantation. Steps toward this end are described later in this
preamble.
C. The Department's Response and Policies of the Final Rule
Because most of the original commenters referenced specific
sections of the NPRM, these comments are generally identified in
numerical terms, e.g., two commenters had suggestions regarding the
definition of ``national list.'' Most subsequent comments, particularly
those made in connection with the public hearing, did not reference the
NPRM. However, most of the latter comments focused on specific issues
(organ donation, organ allocation, liver allocation, and oversight
procedures) and are addressed in the corresponding sections below.
1. Section 121.2--Definitions
``National list'': Two commenters said that the proposed definition
is misleading in that it implies a single, nationwide list for
allocating organs whereas the OPTN policies for allocating organs give
considerable weight to local and regional geographical considerations.
The Department agrees that the term ``national list'' has been used in
conjunction with allocation criteria that involve geographic factors.
However, all recipients of organs are selected from a set of national
databases; and even the current allocation criteria have important
national elements for some organs. Therefore, the Department has
retained the term ``national list.''
``OPTN computer match program'': The Department received two
comments on this definition and has modified it to provide a better
description of the matching process. The new definition states that the
``OPTN computer match program'' means a set of computer-based
instructions that compares data on a cadaveric organ donor with data on
transplant candidates on the national list and ranks the candidates
according to OPTN policies to determine the priority for allocating the
donor organ(s).
``Organ'': The proposed rule defines ``organ'' as a human kidney,
liver, heart, lung, or pancreas. Four commenters suggested that the
definition be broadened to include parts of organs and other organs.
The inclusion of other organs, such as the stomach and intestines, not
only would have an impact on other requirements in these regulations
such as the development of allocation policies, certification of
designated transplant programs, and establishment of training
requirements but also would affect OPO requirements to procure these
organs in accordance with rules of the Health Care Financing
Administration (HCFA). Thus, the Department believes it would be
premature for this rule to specify other organs in addition to those
already named. Instead, the Department will direct the OPTN contractor
to consider
[[Page 16306]]
which organs or parts of organs, if any, should be subject to OPTN
policies, and to submit recommendations to the Secretary. The
Department has added a reference to bone marrow to the definition,
because section 374(d)(1) of the Act provides that the term includes
bone marrow for purposes of the Scientific Registry.
``Organ donor'': One commenter suggested the addition of a
definition for this term. The Department has accepted the suggestion
and has defined ``Organ donor'' as a human being who is the source of
an organ for transplantation into another human being.
``Potential transplant recipient'': The Department has edited this
definition in accordance with the two comments it received. The new
definition more accurately describes the relationship of the individual
to the OPTN computer match program.
``Transplant candidate'': One commenter suggested a broader
definition that the Department has accepted. It now defines
``transplant candidate'' as an individual who has been identified as
medically suited to benefit from an organ transplant and has been
placed on the national list by the individual's transplant program.
``Transplant physician'' and ``transplant surgeon'': The Department
has added definitions for these terms in response to a commenter's
suggestion that they be included. The final rule defines ``transplant
physician'' as a physician who provides non-surgical care and treatment
to transplant patients before and after transplant, and ``transplant
surgeon'' as a physician who actually does transplants and provides
surgical care and treatment to transplant recipients.
``Transplant program'': As suggested by one commenter, the
Department has made an editorial change in this definition.
2. Section 121.3--The OPTN
This section of the proposed rule (originally titled
``composition'') elicited the most written comments, the majority of
which discussed representation on the OPTN Board of Directors and
committees. In addition, the public hearing identified the governance
of the OPTN, including the composition of the OPTN Board of Directors
and committees, as a significant area of concern. OPTN membership is
summarized in Table 2 below.
Table 2--OPTN Membership, 1996
Transplant Centers............................................... 281
Consortium Members............................................... 4
Organ Procurement Organizations.................................. *54
Histocompatibility Laboratories.................................. 55
Voluntary Health Organizations................................... 12
Medical/Scientific Organizations................................. 29
General Public Members........................................... 8
------
TOTAL............................................................ 443
*This only includes independent OPOs; the other 9 OPOs are represented
through their hospitals.
Source: 1996 Annual Report of the OPTN, page C-2 Table C-2.
Both in the written comments and at the public hearings, numerous
witnesses who disagreed on particular organ allocation issues
nonetheless agreed that there is a potential conflict of interest if
transplant professionals, representing particular programs that provide
them employment, vote on matters that may substantially affect the
financial viability of those programs. Others argued that disagreements
among transplant professionals overwhelmingly reflect honest
differences of opinion and the natural desire of physicians and others
to ensure the best possible outcomes for their own patients.
Additionally, the Department received comments regarding the
independence of the process for selecting members of the OPTN Board of
Directors. Some members are currently elected from lists of persons
selected by the nominating committee of the Board of Directors, not
through independent nomination or election by sponsoring organizations.
Regardless of the precise procedures and categories, many people
believe that the OPTN Board of Directors would be more effective and
have enhanced credibility if a greater percentage of its members were
persons who broadly represent the public interest and persons who
directly represent patient interests, without direct employment or
similar ties to the field of transplantation.
The Secretary believes none of the changes being made in the
regulatory provisions describing the composition of the Board of
Directors will jeopardize either the expertise or the continuity of
leadership important to the functioning of the OPTN. Transplant
professionals will continue to be strongly represented on the Board.
However, the rule will foster a broader range of diverse and
independent views.
Accordingly, the Secretary is requiring the following changes in
the composition of the Board of Directors (all in the context of a
Board membership of 30 or more persons, as determined by the OPTN
itself): First, at least eight of the Board members are to be
transplant candidates, transplant recipients, organ donors, or family
members and none of these members or general public members may have an
employment or similar relationship with the OPTN or with the categories
of members listed in Sec. 121.3(a)(1)(I) or (iii)--OPOs, transplant
hospitals, etc. Second, at least six members of the Board of Directors
are to represent the general public; these members must be free of an
employment or similar relationship to the OPTN or institutions or
individuals involved in transplantation. Third, not more than 50
percent of the Board members, and of the Executive Committee, may be
transplant physicians and transplant surgeons. Fourth, at least 25
percent of the Board members must be transplant candidates, transplant
recipients, organ donors, and family members of any of these
categories.
To give the OPTN some flexibility in meeting this new requirement,
the Secretary is eliminating the originally proposed requirement that
every OPTN region be represented on the Board. The Department does not
require even that the OPTN use a regional structure. Thus, no reason
exists to impose regulatory requirements for regional membership on the
Board even if the OPTN continues to use a regional structure on its own
volition.
This will also give the OPTN more flexibility in determining Board
size. Depending on the OPTN's decisions as to size of the Board and
whether the OPTN wishes to have any other members serve in a dual
capacity and represent regions, this could free up as many as 11 seats
on the Board of Directors. For the same reason, the rule gives the OPTN
flexibility in the size of the Board of Directors--making clear that
the contracting organization is free to have its own governing board
structure that is separate and distinct from the structure of the OPTN
itself. The rule gives the OPTN six months from its effective date to
make these changes.
Turning to the original written comments on specific regulatory
language, two comments indicated that the regulatory language in
proposed Sec. 121.3(a)(1) was confusing with respect to the number of
individuals comprising the Board of Directors. The Department agrees
and has not set any requirements as to maximum board size (although the
minimum numbers specified for required members add up to 30 persons).
At present, the Board has 39 members.
Several commenters suggested that patient groups should be
permitted to select their own representatives to the Board and that the
interests of patients and families of patients should be better
represented on the Board and on its
[[Page 16307]]
Executive Committee. The Department agrees with the comments on the
need to ensure that the interests of patients and their families are
represented; however, the Department believes the OPTN should have
flexibility as to its nomination and selection process. Thus,
Sec. 121.3 now provides that eight transplant candidates, transplant
recipients, organ donors, or family members shall be included on the
Board.
In addition, the Department has added to Sec. 121.3 a requirement
that the Board include at least 25 percent transplant candidates,
transplant recipients, organ donors, and family members. Over the last
few years, these individuals have represented 20 to 33 percent of the
Board; and the Secretary expects that a comparable representation will
be maintained. Section 121.3(b)(1) now requires the Executive Committee
to include at least one member who is a transplant candidate,
transplant recipient, organ donor, or family member, one general public
member, and one OPO representative. Section 121.3(b)(3) requires
transplant candidate, transplant recipient, organ donor, or family
member representation on all committees established by the OPTN and
also requires representation by transplant coordinators, OPOs, and
transplant hospitals, as suggested by several commenters. The
Department expects the OPTN to determine the appropriate number of such
representatives on each committee, based on the types of issues that
the committee will address.
The American Society of Transplant Physicians (ASTP) commented that
it should select its own Board representative. The Department disagrees
that it would be useful to add such a requirement, because transplant
physicians are otherwise well represented on the Board and those
members are members of the ASTP.
Another individual commented that the Board should include more
minority representation. Proposed Sec. 121.3(a)(2)(i) requires that the
Board of Directors include individuals representing the diversity of
the population of organ donors and recipients served by the OPTN,
including minority and gender representation reflecting that diversity.
A similar requirement with respect to committees is proposed at
Sec. 121.3(b). The Department has reviewed these proposed requirements,
considered the commenter's suggestion, and decided to clarify these
requirements in the final rule. The Department believes that including
individuals from groups under-represented in the transplant patient
population would enhance the ability of the OPTN Board and its
committees to address the critical health needs of these populations.
However, because the Board is elected, its composition is not
guaranteed to reflect minority and gender diversity. Moreover, the
Department intended that the Board requirement parallel the requirement
for committees, that is, that the OPTN should attempt to reflect such
diversity ``to the extent practicable.'' In neither case, however, does
the Department intend to impose requirements that it would enforce,
although, the Department strongly urges the OPTN to consider
appropriate and practicable ways to encourage participation by
minorities and women on its Board and on its committees.
One commenter asked that the general public category be broadened
to include ``pre-transplant'' patients. As proposed,
Sec. 121.3(a)(1)(ii)(F) lists examples of individuals who could be
elected from the general public. Because the section also says that the
general public category is not limited to the examples given, ``pre-
transplant'' patients could be chosen. However, the Department has
modified Sec. 121.3(a)(1), as discussed above, by adding the category
transplant candidates, transplant recipients, organ donors, and family
members to Sec. 121.3(a)(1)(ii). This addresses the interests of
transplant patients and candidates (pre-transplant patients), and
transplant recipients, as well as family members of individuals who
have donated or received an organ. Also, transplant candidates now are
included within the diversity requirements of Secs. 121.3(a)(3)(i) and
121.3(b)(3)(ii).
Another commenter suggested that regional representatives to the
Board be elected from OPOs rather than transplant hospitals. The NPRM
does not identify an organizational affiliation for regional
representatives, nor does the final rule. Thus, regional
representatives, if the OPTN elects to continue this approach, may be
individuals affiliated with OPOs. They could also include other
individuals who are affiliated neither with OPOs nor with transplant
hospitals.
Two other commenters recommended staggered terms for Board members.
One commenter recommended that the Executive Committee be elected
annually rather than every two years as proposed; and three commenters
said that proposed Sec. 121.3(a)(5), requiring the appointment of an
Executive Director to serve a four-year term, was unnecessary. We agree
and have deleted that requirement. The existing OPTN practice is to
stagger the terms of Board members, and the Department believes that
the OPTN will continue to manage this aspect of its operation without
the need for Federal regulation. With respect to annual election of the
Executive Committee, the Department sees no reason to impose this
requirement. In sum, we have tried to specify only the most essential
features of the OPTN governance structure and to give the OPTN maximum
flexibility in making decisions on other aspects of governance.
Two commenters said that all of the policy development duties of
the Board of Directors in proposed Sec. 121.3(a)(6) should be subject
to the public participation process in proposed Sec. 121.7(b),
requiring public comment on proposed organ allocation policies. As
mentioned above, we have added a new Sec. 121.4 to clarify the intent
of the policy development processes in the proposed rule. New
Sec. 121.4 incorporates the regulatory language in proposed
Sec. 121.3(a)(6) concerning the development of policies by the OPTN
Board of Directors, the regulatory language of proposed Sec. 121.7(b)
regarding the public participation and appeals processes required for
policies, and the regulatory language of proposed Sec. 121.10 on review
and appeal of policies.
Proposed Sec. 121.3(a)(6)(ii) requires that the OPTN provide to the
Secretary copies of all policies as they are adopted and make them
available to the public upon request. It also states that the Secretary
will periodically publish lists of these documents in the Federal
Register. The Department has retained these requirements in new
Sec. 121.4(c) and has added a requirement that the Board of Directors
provide the OPTN membership with copies of the policies (as well as
notification of upcoming Board meetings). In addition, the Secretary
will publish a statement indicating which OPTN policies trigger the
special compliance requirements and potential sanctions under section
1138 of the Social Security Act.
The Secretary also has added a requirement that copies of all OPTN
policies be continuously maintained on the Internet, to provide access
to OPTN members, patients, donor families, transplant professionals,
and other persons interested in organ transplantation. (The OPTN
already operates an extensive and valuable Web site that substantially
meets this requirement, at http://www.unos.org.) All policies of the
OPTN are subject to review by the Secretary at any time under
Sec. 121.4(b)(2) and policies may be appealed under Sec. 121.4(d). The
Secretary will determine which policies should be subject to the notice
and
[[Page 16308]]
comment process of the Administrative Procedure Act.
An editorial change was suggested to delete from proposed
Sec. 121.3(a)(6)(i)(B) the words ``fair and'' from the phrase ``fair
and equitable allocation of human donor organs.'' The Department agrees
that the proposed language is redundant and has accepted the
recommendation. See Sec. 121.4(a)(1).
With respect to the proposed requirements for OPTN membership,
several commenters suggested that the rules establish voting and non-
voting membership categories or otherwise set out membership voting
privileges. The Department believes this is appropriate for the OPTN's
policy development process and expects the OPTN to submit to the
Secretary for review policies it has already developed in this regard.
Two commenters pointed out what they perceived to be a drafting error
in proposed Sec. 121.3(c), which states that the OPTN shall admit and
retain as members organizations, institutions, or individuals that have
an interest in organ transplantation. The commenters said that the word
``shall'' should be changed to ``may'' to give the OPTN discretion in
granting membership under Sec. 121.3(c)(3). The Department has retained
the mandatory term ``shall'' because we believe that anyone with a
documented interest in organ procurement and transplantation must be
granted membership. Should the OPTN deny membership under
Sec. 121.3(c)(3), applicants may appeal to the Secretary under
Sec. 121.3(c)(4). In addition, we have added to Sec. 121.3(c)(3) a
requirement that the OPTN process membership applications within 90
days to establish in principle that the Secretary expects the process
to be carried out as expeditiously as possible given the OPTN's
operational constraints.
The Secretary has added a new subsection 121.3(d) on corporate
status of the OPTN. That section recognizes that requirements as to
composition of the Board of Directors and membership admission
requirements could create some problems for the OPTN contractor. The
current contractor, a Virginia corporation, has chosen to recognize
OPTN membership as automatically creating a right to corporate
membership. At some future time, this or some other contractor might
wish to create different arrangements. The language in this rule allows
for this and clarifies that OPTN members do not have to become (nor the
contracting corporation to accept them as) members of the corporation.
The Secretary has also added a provision at Sec. 121.3(e) that allows
current and future contractors six months to come into full compliance
with regulatory requirements in this section.
3. Section 121.5--Listing Requirements (Formerly Sec. 121.4)
Most of the original comments received on this section of the
proposed rule were on the subject of multiple listing, either
supporting or opposing it. The proposed rule, in keeping with existing
policy, did not prohibit transplant candidates from being listed with
more than one transplant hospital. The final rule adopts this policy
despite the commenters' concerns that it may disadvantage individuals
who lack the insurance coverage or resources to seek listing with more
than one institution or may raise ethical issues.
The Department believes that multiple listing is one of the few
avenues open to patients who wish to choose their own medical care
providers or try to overcome the waiting time inequities produced by
the current ``local first'' allocation policies. Moreover, under
current allocation policies, multiple listing helps patients who prefer
to use a nearby transplant hospital that falls outside the so-called
``local area'' instead of a distant hospital that falls within that
boundary. In addition, very few patients select this option. Steps to
reduce waiting time inequities are described later in this preamble.
When waiting times have become substantially equivalent among programs,
the Secretary may ask the OPTN contractor to revisit the issue through
its policy development process and submit its recommendations to the
Secretary.
Several commenters suggested replacing the term ``OPTN member'' in
proposed Sec. 121.4(a)(1) and (3) with ``transplant hospital.'' The
Department has accepted the suggestion with respect to proposed
Sec. 121.4(a)(1). See, Sec. 121.5(a). However, because registration
fees may be paid by OPTN members other than transplant hospitals, we
have not made the suggested change in proposed Sec. 121.4(a)(3). See,
Sec. 121.5(c).
Several commenters said that a time limit should apply when the
OPTN submits to the Secretary a request for approval of the
registration (listing) fee. The Department agrees in principle that
such requests should be handled promptly and has added a requirement
that the Secretary will approve or disapprove the amount of the fee
within ``a reasonable time'' of receiving a request for approval and
such supporting information as will provide the Secretary an informed
basis for that decision. See, Sec. 121.5(c). This language allows for
the Secretary's discretion to publish a notice requesting public
comments on any change in the registration fee. If the necessary
supporting information is provided, a ``reasonable time'' should not
exceed 30 days, and the Department will make every effort to meet that
deadline. We welcome suggestions as to whether additional steps are
needed to ensure that OPTN revenues are properly used for OPTN
purposes.
One commenter suggested adding a new section requiring transplant
hospitals to provide patient acceptance criteria to all patients. The
Department agrees that patients should have access to as much
information as possible. However, such a requirement would be very
difficult to craft and enforce and would involve providing detailed
medical information, because acceptance criteria are based on the
varying medical conditions associated with end stage organ failure.
Instead of creating a specific provision, we are greatly strengthening
various requirements (see below) related to disclosure of information
of benefit to patients.
4. Sec. 121.6--Organ Procurement (Formerly Sec. 121.5)
All but one of the comments received on this section concerned the
criteria for acceptance of donor organs. Proposed Sec. 121.5(c) permits
transplant programs to establish such criteria but does not require it.
Suggestions ranged from requiring minimum acceptance criteria to
establishing standardized or universal criteria. The Department agrees
that criteria are necessary and has added a requirement for the
establishment of criteria for organ acceptance. See, Sec. 121.6(c).
However, we defer to the OPTN on whether to establish standardized
criteria. Should the OPTN decide that such criteria are desirable, we
expect such a decision, as well as the criteria themselves, to be
developed through Sec. 121.4, discussed above.
5. Section 121.7--Identification of Organ Recipient (Formerly
Sec. 121.6)
This section of the proposed regulations (formerly Sec. 121.6)
prompted a number of editorial suggestions, as well as concerns about
financial responsibility for the transport of donated organs and
protecting the confidentiality of organ donor records. The Department
has accepted the editorial suggestions. One commenter said that
proposed Sec. 121.6(a)(4) should include a requirement that the OPTN be
advised of the reasons for a transplant hospital's refusal of an
offered organ. The Department agrees with this
[[Page 16309]]
suggestion, which is consistent with current practice, and has included
it. This notice is to go to the hospital's affiliated OPO as well. See,
Sec. 121.7(b)(4).
Several commenters expressed concern about protection of
confidentiality of donor records required by proposed Sec. 121.6(c)(2).
The Department agrees that such records must be protected and is
confident that adequate safeguards exist in Federal and State
legislation. No specific provisions are required in this regulation.
According to two commenters, proposed Sec. 121.6(c)(1) should be
amended to indicate that either a transplant hospital or an OPO is
responsible for transporting a donated organ. Another suggested setting
limits on, or otherwise accounting for, the financial implications of
``unreasonable'' transport requests. The Department intended that
proposed Sec. 121.6(c)(1) be broad enough to allow for a variety of
situations that could arise in the transport of a donated organ.
Moreover, proposed Sec. 121.6(c) does not assign financial
responsibility for such arrangements, which, with respect to
transplants reimbursed by Medicare and Medicaid, are within the purview
of HCFA and its regulations related to organ acquisition costs.
Three commenters said that OPOs cannot ensure the viability of
transported organs, as indicated in proposed Sec. 121.6(c)(3). The
Department agrees and has modified this paragraph to require that the
OPTN members transporting an organ ensure that it is packaged to
enhance the probability that the organs will remain viable. See,
Sec. 121.7(c)(3).
Proposed Sec. 121.6(d) elicited several comments pointing out that,
in practice, OPOs make the offer of donor organs, not transplant
hospitals. The Department agrees and has modified the language to
delete the reference to transplant hospitals. See, Sec. 121.7(b). We
have also changed the term ``OPTN member'' in proposed Sec. 121.6(e) to
``transplant hospital'', as suggested by one commenter. See,
Sec. 121.7(e).
6. Section 121.4--Policies: Secretarial Review (Formerly Sec. 121.7(b)
Public Participation)
Based primarily on the issues raised at the public hearing, this
section has been expanded to include a new requirement
(Sec. 121.4(a)(3)) that the OPTN modify or issue policies to reduce
inequities resulting from socioeconomic status to help patients in need
of a transplant be listed and obtain transplants without regard to
ability to pay or source of payment. While such access is not
guaranteed for other medical procedures, transplantation presents a
special case. Donation is a valuable gift that is not conditioned on
ability of recipients to pay nor do donors pass a ``means'' test. For
these reasons, further efforts to facilitate access to the ``gift of
life'' are necessary.
The Secretary does not prescribe specific steps, but requires the
OPTN to consider possible policies to reduce inequities. For example,
the Secretary expects the OPTN to consider methods of waiving or
financing listing fees for patients unable to pay, through some form of
cross-subsidy or by requiring that member hospitals absorb such fees.
The problem of paying for the transplant itself is much more
complex, given the cost of these procedures, but a number of
possibilities exist. Many member hospitals, for example, are obligated
to provide uncompensated care under their charters or through the Hill-
Burton requirements imposed as a condition of public grants and
subsidized loans. The OPTN directly, or through member hospitals, could
seek charitable contributions. Member hospitals could be obliged to
provide a certain fraction of their transplants without charge to the
patient, in recognition of the substantial value of the ``gift of
life'' that the donors and families have provided for purely altruistic
motives. Medicaid reimbursement could be sought more aggressively, for
example, through the ``spend down'' provisions that enable many persons
to qualify for insurance under that program. These and other options
present difficult problems of policy and design; the Secretary simply
requires here that the OPTN devote its energy to devising solutions and
proposing policies to implement them. We are particularly interested in
ideas that the OPTN could use to implement this provision.
As previously discussed, this general subject consumed a great deal
of time and attention at the public hearings. Those hearings did not,
however, focus on the details of the proposed rule or on how best to
amend those.
With respect to proposed Sec. 121.7(b), the Department received
three comments during the original comment period about the process of
adopting final allocation policies. Two commenters raised the issue of
publishing proposed changes to allocation policies in the Federal
Register. One said that the Secretary's decisions should be published;
and the other suggested that, to meet the requirements of the
Administrative Procedure Act, all proposed changes should be published
with analyses before the Secretary makes a decision.
UNOS asked if the OPTN contractor would be required to submit to
the Secretary for approval allocation policies in effect on the
effective date of the final rule, pursuant to the process described in
the final rule. For policies that the OPTN wants to be enforceable, the
answer is yes. With the exception of particular policies established in
this rule, all policies that have not been approved by the Secretary as
enforceable remain voluntary, as explained in the 1989 Federal Register
Notice. OPTN members that disagree with those policies may appeal them
to the Secretary.
During the public hearing, a great many comments were directed to
the question of the appropriate level of Federal oversight. While
virtually all commenters agreed that the Department should have some
role, opinions as to what that role should be varied from passive
monitoring to taking very direct charge. Many of the particular
suggestions made reflected the legal constraints that apply to organ
transplantation. Some of these commenters also misunderstood the role
and obligations of the Federal government for requirements that are
established by law, even if implemented in part through private parties
rather than by Federal staff. If the OPTN were a purely voluntary
organization that happened to be a Federal contractor and if approved
OPTN rules had no binding effect on patients or hospitals, then the
appropriate level of oversight might be relatively low and limited
primarily to efficient execution of the contract. But under the current
law, patients have, as a practical matter, no choice but to use the
system governed by the OPTN. Moreover, hospitals can lose the right to
participate in Medicare and Medicaid and OPOs can lose reimbursement
under Medicare and Medicaid for noncompliance with OPTN rules and
requirements.
Both the genesis and wording of the National Organ Transplant Act
(NOTA), as amended, obligate the Secretary to utilize the
transplantation community substantially in both developing and
executing transplantation policy. Under the statutory framework
established by the Congress, however, the Department has oversight
obligations, arising from the NOTA, as well as other laws and executive
orders. For example, the Secretary has an affirmative obligation to
make sure that policies and actions of the OPTN do not violate the
civil rights of candidates for organ transplants. In this regard,
however, most commenters stated, and the Secretary agrees, that
[[Page 16310]]
Departmental oversight should not micro-manage the development of
purely medical criteria or routine day to day decision-making of
attending medical professionals or the OPTN contractor.
The Department, in the preamble to the proposed rule (59 FR 46486),
made clear its intention to provide the public with an opportunity to
comment on organ allocation policies and proposed changes to them.
While we believe that the comment process administered by the OPTN
itself is invaluable in obtaining technical advice, it does not reach
all of the affected public--including potential donors and interested
persons who are not OPTN members and have no access to the OPTN--or
otherwise provide the functions and protections accorded by the
impartial review by the Secretary. These principles are carried forward
in the final rule. To allow sufficient time for public comment on
policies that the Secretary decides to publish, we have deleted from
proposed Sec. 121.7(b)(3) the 30-day time limitation and have
substituted ``within a reasonable time.'' See, Sec. 121.4(c)(2). The
Secretary recognizes the importance of these issues, and expects the
Department to act expeditiously on them. To ensure stability of the
system, organ allocation policies, once implemented, continue to be in
force during pending appeals or revisions.
New Sec. 121.4 provides for an ongoing process of review that
attempts to marry several goals: relying on the expert OPTN process to
the maximum extent feasible; providing for independent review by the
Department with additional opportunity for public comment; providing
for cases where changes in policies may need to be made more rapidly
than either process or both together would allow; and allowing the
Secretary to take such other actions as the Secretary deems
appropriate. Key to the effective functioning of this process is the
acceptance by the transplant community of OPTN policies that have not
been (and may never be) formally approved as enforceable requirements,
but that most institutions choose to accept. A body of voluntary
standards that can be rapidly revised, particularly for purely
technical changes, is a crucial function of the OPTN system and one
that the Secretary strongly supports. The Secretary believes that this
rule puts in place an approach that accommodates all of the above
goals.
7. Section 121.8--Allocation of organs
The majority of written comments received on proposed Sec. 121.7
were opinions both for and against elements of the existing individual
organ allocation policies, rather than comments on the content of this
section of the proposed rule.
Several people discussed either the desirability or undesirability
of permitting variances to current policies for allocating organs.
Other commenters suggested broadening the geographic areas for organ
allocation, localizing the areas for organ allocation, or allocating
organs on a nationwide basis. One commenter said that allocation should
be nationwide, because the current system is unfair to veterans. Under
the medical coverage provided by the Department of Veterans Affairs
(VA), veterans who need organ transplants are required by the VA to be
listed with a transplant program with which the VA has contracted.
Another commenter said that local allocation is an important incentive
to organ procurement and that the relationship should be studied.
Another commenter objected to disparities in waiting time among
geographic areas.
The American Society of Transplant Physicians suggested a
conference to determine the suitability of patients for transplant, the
establishment of standardized criteria to determine when a patient
should be placed on the waiting list, and to define standards for a
patient to be retransplanted. The United Network for Organ Sharing
(UNOS), the OPTN contractor, provided a list of factors to be
considered by the OPTN Board of Directors in developing organ
allocation policies. All of these issues are addressed in this
preamble. The Secretary notes that since the publication of the NPRM,
some of these suggestions have been adopted.
The Secretary originally received 62 letters commenting on organ
allocation policies, of which 50 were about the lung allocation policy
(many of those concerning lungs were form letters from patients at a
single institution). These commenters, most of whom were individuals
identifying themselves as organ transplant recipients, potential
recipients, and friends or relatives of potential recipients, urged
that geographic areas for lung allocation be broadened to permit more
organs to be allocated to a particular medical program.
Comments on other organ allocation policies were also received from
individuals affiliated with hospitals, from the American Society of
Transplant Physicians, from the Cystic Fibrosis Foundation, from a law
firm representing a hospital, and from a member of Congress on behalf
of a constituent. Two comments were on the kidney allocation policy,
one supporting local allocation and the other providing a copy of
technical comments sent to the OPTN on revising the point system. One
comment was on the heart allocation policy, suggesting that the
geographic boundaries for allocation under the current policy be made
more flexible. Two comments were not specific with respect to a
particular organ, but recommended that allocation be nationwide based
on time on the waiting list.
The Secretary also received letters urging action on liver
allocation with emphasis on wider sharing. These comments, and many
others on related allocation issues, arising both in the original
comment period and at the public hearing, are addressed below in our
proposed performance goals.
When the proposed rule was issued in 1994, the Department posed
several open-ended questions about allocation policy, with the
expectation that public response would help us decide how best to
handle allocation policy and the extent to which we would seek to
establish such policy in this final rule or in policy-by-policy
reviews. Both in the initial set of public comments and in the months
surrounding the public hearing, the Department received a great deal of
information about, and many criticisms of, current allocation policies.
For example, we learned that current allocation policies, by allowing
local geographic boundaries to override patient needs, do not follow an
ethical opinion addressing this very issue, promulgated through the
Code of Medical Ethics of the Council on Ethical and Judicial Affairs
of the American Medical Association. Second, we received the early
results of computer modeling sponsored independently by UNOS and the
University of Pittsburgh Medical Center (UPMC). These modeling efforts
provided quantitative estimates of a great many variables--lives saved
both pre-and post-transplant, time on waiting list, graft survival
rates, etc.--that had previously been difficult to address
systematically when alternative allocation policies were compared.
Third, the OPTN itself continued to study, debate, and consider major
revisions to its policies. Building on this new information, a primary
purpose of the December hearings was to obtain even more information
and opinions on organ allocation policies, particularly those affecting
livers. That purpose was achieved.
Based on these sources and much other information, the Department
has determined that the original proposal in the NPRM was insufficient.
The
[[Page 16311]]
transplantation community is very divided, on allocation policy in
general and specifically on liver allocation, and the existing policy
development process is unlikely to bridge those divisions. Medical
issues, ethical issues, and matters of trust and actual practice are
substantially intertwined. Yet, the Department is unwilling, at this
time, to issue a prescriptive allocation policy. We believe the OPTN
must be primarily responsible for establishing medical criteria for
patient listing and status categories, and for developing equitable
allocation policies that reflect the Secretary's policies, as expressed
in this regulation.
The Secretary decided, therefore, to approach the issue in terms of
performance goals. The basic idea of a performance goal is to set a
target, allow the operating entity (in this case the OPTN) to determine
how best to meet that goal, and then measure performance against that
goal. This model is widely used in business and in public programs. It
is the model for this Department's Healthy People 2000 goals and other
initiatives as well as the recently enacted Government Performance and
Results Act. Quite apart from its other advantages, it promises to
clarify and strengthen the Department's review and approval process for
OPTN policies.
Based on the detailed and helpful dialogue at the hearing, and the
clearly expressed preferences of commenters on both sides of specific
issues, the Secretary has determined that three broad performance goals
for organ allocation are needed. The topics of these goals are: (1)
minimum listing criteria, (2) patient status, and (3) priority for
patients with the highest medical urgency. The Secretary has also added
a requirement, discussed below, for the OPTN to assess the cumulative
effect of its policies, and develop new policies as appropriate,
regarding socioeconomic equity. All of these goals are subject to sound
medical judgment, both as to specific patients and as to overall
standards, in order to avoid organ wastage, reflect advances in
technology, and otherwise operate an effective and efficient allocation
system.
Listing (Sec. 121.8(a)(1)). Many commenters at the hearings pointed
out that current allocation policies (which give substantial weight to
overall waiting time without regard to status) encourage aggressive
physicians to list patients for transplants as early as possible, in
some cases years before they will need or want a transplant. Other
physicians are more conservative, and some patients do not come to the
attention of transplant professionals until later in the course of
their underlying condition. As a result, persons with equal waiting
times may have very different medical urgency. This means that overall
waiting time as a ``tie-breaker'' is unfair, encourages ``gaming''
behaviors and distrust within the transplant community, and discourages
sharing of organs across geographic areas (because a less needy patient
in one local area may obtain preference over a more needy patient in
another local area simply by virtue of aggressive early listing). We
have determined, therefore, to require that the OPTN develop listing
criteria that are based on objective medical criteria pertinent to each
organ, and to update these criteria to reflect increasing medical
knowledge. The OPTN already has efforts underway that go a long way
toward achieving this objective, and the Secretary applauds those. As
explained below, overall waiting time will also be replaced by waiting
time in status as a ``tie breaker.''
Patient Status (Sec. 121.8(a)(2)). Another set of themes emerging
from the hearings is the recognition that current liver allocation
criteria fail to differentiate adequately among different degrees of
medical urgency and the desire for substantial improvements in the use
of objective medical criteria for the classification of patients. In
some cases, existing criteria are based on situational factors, such as
whether a person is hospitalized, which are neither medical criteria
nor necessarily good proxies for underlying medical condition or
urgency. They can also encourage choices on the part of managing
physicians to make sure that their own patients are not disadvantaged
relative to other persons. At the same time, we know that advances in
transplantation medicine and the OPTN's extensive investment in patient
information systems have made possible improvements in the
classification of patients. The ever-improving knowledge base about the
medical factors that correlate with transplant outcomes, combined with
the use of computer technology and statistical analysis, allow
sophisticated ranking of patients, without the need to group disparate
patients into relatively few and crude categories. The Secretary has
decided to endorse the requested reforms and require improved
categorization of patients, based on objective medical criteria that
distinguish among different levels of urgency in sufficient detail as
to reduce discriminatory effects.
Priority for the Most Urgent and Geographic Equity
(Sec. 121.8(a)(3)). By far the most controversial aspect of current
allocation policies is that the ``local first'' feature creates
inequities in access for organs among patients of equal medical
urgency, making where they live or list a more important factor than
objective measures of medical status in obtaining an organ. All
patients are affected by these inequities, but the consequences fall
most heavily on those whose medical need is greatest and who are most
likely to die before receiving an organ. As shown in tables 3a and 3b
below, there are vast differences in median waiting times for kidneys
among different transplant programs and different organ procurement
areas (table 3a addresses transplant hospitals and is adapted from OPTN
data printed in the Cleveland Plain Dealer on February 5, 1997; table
3b addresses organ procurement areas and is adapted from OPTN data on
waiting times that will shortly be published):
Table 3a.--Shortest and Longest Waiting Times by Kidney Transplant
Program 1994-1995
------------------------------------------------------------------------
Median
------------------------------------------------------------------\1\---
Shortest Hospital Waiting Times:
Harris Methodist, Fort Worth, TX........................... 54
Presbyterian-University, Pittsburgh, PA.................... 79
Southwest Florida, Fort Myers, FL.......................... 114
Henrietta Egleston, Atlanta, GA............................ 144
Oregon Health Sciences, Portland, OR....................... 147
Longest Hospital Waiting Times:
University of Pennsylvania, Philadelphia, PA............... 822
Northwestern Memorial, Chicago, IL......................... 828
Lehigh Valley, Allentown, PA............................... 838
William Beaumont, Royal Oak, MI............................ 850
Milton Hershey, Hershey, PA................................ 858
------------------------------------------------------------------------
\1\ Median waiting times (days).
Source: Cleveland Plain Dealer, February 5, 1997, reporting UNOS data.
Table 3b.--Shortest and Longest Kidney Transplant Waiting Times by Local
Allocation (OPO) Area, 1993-1995 for Blood Type O
------------------------------------------------------------------------
Median
------------------------------------------------------------------\1\---
Shortest OPO Waiting Times:
Oregon Health Sciences University Hospital................. 107
Lifelink of Southern Florida............................... 143
Lifelink of Florida........................................ 161
Life Connection of Ohio.................................... 204
Longest OPO Waiting Times:
Carolina Organ Procurement Agency.......................... 1,423
[[Page 16312]]
Regional OPA of Southern California........................ 1,501
California Transplant Donor Network........................ 1,513
New York Organ Donor Network............................... 1,680
------------------------------------------------------------------------
\1\ Waiting times (days).
Source: UNOS data, soon to be published in report on waiting times. The
OPO waiting times are longer than hospital waiting times mainly
because type O patients wait longer than most other blood types.
Unfortunately these data, although the best available, do not
isolate the differences in patient condition or in transplant centers
listing practices that underlie some of the observed disparity. For
example, as discussed previously, some doctors aggressively list
patients very early in the course of their disease to give them more
waiting time and raise their chance of obtaining an organ. Such a
practice artificially inflates waiting times in some areas. However,
the differences in waiting times by area far exceed the differences in
medical status by area.
These differences exist throughout the United States. As shown in
Table 4, each OPTN region has many local OPO allocation areas with
relatively short and relatively long waiting times:
Table 4.--Range of Kidney Transplant Waiting Times Among OPOs by OPTN
Region Median Waiting Time in Days, 1994 for Blood Type O
------------------------------------------------------------------------
Days (shortest-
Median waiting times for kidneys longest)
------------------------------------------------------------------------
Region 1 (New England).............................. 413-1,360
Region 2 (DC, DE, MD, NJ, PA, WV)................... 702-1,378
Region 3 (Southeast)................................ 143-761
Region 4 (OK, TX)................................... 386-655
Region 5 (California & Southwest)................... 374-1,513
Region 6 (Northwest)................................ 107-1,061
Region 7 (Upper Midwest)............................ 794-1,176
Region 8 (CO, IA, KS, MO, NE, WY)................... 287-754
Region 9 (NY)....................................... 228-1,680
Region 10 (IN, MI, OH).............................. 204-1,422
Region 11 (KY, NC, SC, TN, VA)...................... 231-1,423
------------------------------------------------------------------------
Source: UNOS data, soon to be published in report on waiting times.
Similar waiting time differences exist for other organs. To some
degree, these differences in waiting times result from the current
absence of standardized listing criteria, as discussed above. Hence,
these are imperfect measures of differentials. They also reflect,
however, the fact that current patients who happen to list in areas
with either higher incidence of end stage organ disease, or less
ability to generate organ donors, are systematically disadvantaged by
policies that do not permit the organs to go to the patients who need
them the most. They also work to the disadvantage of prudent purchasers
who wish to designate or contract with particularly high quality (or
low cost) transplant hospitals to serve their patients. Under current
allocation policies, neither individual patients nor concerned payers
have the freedom to select their preferred medical provider without, in
many cases, increasing the chance that the patient will wait longer and
die while waiting for an organ.
Individual patients are directly affected, regardless of medical
need. Although the Department is mindful that anecdotes can be
misleading, the following example illustrates the inherent effects of
establishing unduly restrictive geographic barriers to equitable organ
allocation. In a recent case reported in the press (Sunday World Herald
of Omaha, Nebraska, May 25, 1997), a patient was forced to choose
between listing with a ``local'' hospital 250 miles away but in an
organ procurement area that covered his State and had access to
relatively more organs, or with his strongly preferred and truly local
hospital just 20 minutes across a river and in another State that had
access to relatively fewer organs. Cases such as this are inherent in a
system that established defined areas for the purposes of administering
organ procurement, but whose boundaries also have been used to limit
organ allocation. Reliance on boundaries that make sense for
administrative convenience may lead to inequities in organ allocation
criteria. For example, in a number of States one OPO is surrounded by
another; and in Texas there is an OPO that is composed of four non-
contiguous areas separated by other OPOs. Some OPOs are based on the
service area of a single hospital; some follow the boundaries of a
single State; and others serve four or more States. These and other
vagaries of this system are shown in the following map. Because of the
differences in OPO size, geography, and population, the Secretary has
decided that OPO areas should not be the primary vehicle for organ
allocation.
BILLING CODE 4160-15-P
[[Page 16313]]
[GRAPHIC] [TIFF OMITTED] TR02AP98.000
BILLING CODE 4160-15-C
[[Page 16314]]
Payers are also directly affected. Their ability to select
transplant hospitals for their patients is hampered if listing patients
solely at those hospitals forces them to compete with local patients
for the limited supply of local organs, even though this listing frees
up organs in the areas in which the patient would otherwise be listed.
Some large payers have tools at their disposal to ameliorate this
problem, such as listing some patients at more than one center
(multiple listing), listing some patients at centers with shorter
waiting lists, or accelerating hospitalization to put patients in a
preferred status. However, most payers do not use such techniques and
only a minority of patients benefit from such ``gaming.''
Perhaps the greatest inequity that the current system of local
priority creates is that it particularly disadvantages those who face
imminent death through unusually rapid deterioration. The chance that
an organ that will match one's physiology will be available in the
local area within the next week is very small. Yet, the chance that an
appropriate organ will be available somewhere in the country and that
it can be transported without risking wastage is much higher.
The transplant community has differing opinions over the issue of
broader sharing. According to some commenters, this is in part because
some hospitals and their patients reap the benefits of a highly
productive OPO and they are concerned that they may receive fewer
organs under a national system. Many commenters have pointed out that
local preference draws upon, and reinforces, close bonds among local
organ procurement organizations and local hospitals and physicians.
Almost all agree that there are logistical and practical reasons why
organs cannot be shipped back and forth across the country in response
to the daily needs of every individual patient.
As shown below in Table 5, there are great disparities among OPOs
in the production of donor organs, and under the current system, the
productivity of the local OPO directly impacts on the number of
transplants done in the OPO service area.
Table 5.--Donors Per Million Population 1995
------------------------------------------------------------------------
Percentage
Donors per million pop. of OPOs
------------------------------------------------------------------------
<15.00..................................................... 19.4="" 15.00-20.00................................................="" 22.4="" 20.01-25.00................................................="" 37.3="" 25.01="">..................................................... 20.9
------------------------------------------------------------------------
Note: The range of OPO donors per million population is 6.4 to 31.6.
Source: Calculation by the Division of Transplantation using UNOS Data.
Major review agencies, including the Inspector General of this
Department and the Congress' General Accounting Office, have reviewed
allocation issues and issued reports concluding there are major
inequities and that major reform is needed to make the allocation
system a truly ``national'' system as intended by the Congress.
The American Medical Association has studied organ allocation
through a panel of experts. In its 1996 Code of Medical Ethics it
states that: ``Organs should be considered a national, rather than a
local or regional, resource. Geographical priorities in the allocation
of organs should be prohibited except when transportation of organs
would threaten their suitability for transplantation.'' In reaching
this conclusion, the AMA panel reviewed the evidence concerning several
organ types, and a wide range of alternative formulations. Of
particular importance was their finding that current organ allocation
policies were, in some cases, seeking to favor patients of lesser
urgency but more likely to benefit, but that in actual practice these
benefit differences were far too small to justify differential
priority.
Taking all of these arguments into account, the Secretary has
determined that a national performance goal is needed to encourage the
OPTN to take advantage of advances in technology and survival rates,
and to bring policies in line with the intent of the National Organ
Transplant Act. That goal would reduce geographic inequities by
requiring that persons with equal medical urgency (i.e., in the same
status as defined under the second performance goal) have essentially
equal waiting times regardless of where they list. This standard
emphasizes, however, that the sickest categories of patients should
receive as much benefit as feasible under this standard, in accordance
with sound medical judgment. This is a significant departure from
current policies, not only in making geography less important for
allocation purposes, but also in its approach to waiting time
disparities. The relevant ``tie-breaker'' will no longer be total
waiting time, perhaps years, but will become waiting time within a
group of patients with equal medical urgency.
We are mindful that there are practicalities involved, including
especially transportation. The problem is not occasional cross-
continental shipping from one large city to another, which is
relatively straightforward. Instead, however, there can be severe
logistical problems with frequent shipping of organs (often preceded by
a special team that travels to retrieve the organ and return with it),
or with moving organs among relatively transportation-disadvantaged
areas, even within the same State. The performance goals are designed
to allow (and require) the OPTN to craft policies tailored to each
organ transplant type that are workable, feasible, and avoid organ
wastage.
Many commenters urged that the Secretary require national sharing
of organs, without any role for geographic factors. Others urged
regional sharing. We prefer the performance goal approach. Achieving
the goal will certainly require greater geographic sharing and will
probably require national sharing for some organs for patients with
specified medical conditions. Indeed, regional sharing is already a
prominent feature of heart allocation, and national sharing a prominent
feature of kidney allocation. However, we believe that any simple
formulation would inhibit the ability of the OPTN to craft the most
sensible policies that achieve practical as well as ethical results,
and we wish to encourage change over time as medical science and
medical criteria improve. Therefore, we are at this time using the
performance goal approach for all organs (with an accelerated schedule
for the initial revision of policies for liver allocation).
Implicit in the requirement that patients with equal medical
urgency and waiting time in status have an equal chance of receiving an
organ is reform of policies that encourage organs to be diverted from
patients of blood type O, the ``universal donor,'' in favor of patients
of other blood types, if that would preclude equalization of waiting
times in status. One of the inequities of present organ allocation
policies is that patients of blood type O wait much longer for organs
than other patients. For example, according to recently calculated data
from the OPTN, the median waiting time for primary kidney transplants
in 1994 was 824 days overall, but 1,007 days for patients of blood type
O. For hearts, the median waiting time was 224 days overall, but 353
days for patients of blood type O in
[[Page 16315]]
1996. Blood type is not an indicator of medical urgency, although it is
a key determinant in organ matching.
The Secretary appreciates that there are many factors that can
contribute to achieving the geographic equity goal. For example, if the
Department's organ donation initiative were to achieve a high rate of
success, then fewer organs would need to be shared. Improved listing
criteria and medical status categories will reduce measured inequities.
Nonetheless, within foreseeable parameters, we see no basis to expect
that inequities can be eliminated for any major organ category without
broader geographic organ sharing, on at least a broad regional basis
for all patients with high levels of urgency.
We also require the OPTN to take into account key constraints on
organ allocation. There are patients with urgent need for whom
transplantation is futile. Organs cannot be used without an assessment
of the immune system and other physical conditions of patients. Broad
geographic sharing should not come at the expense of wasting organs
through excessive transportation times. Efficient management of organ
allocation will sometimes dictate less transportation when the highest
ranking patient can wait a day or two for the next available organ.
Sound medical judgment must be exercised before a final decision on
whether to transplant a particular organ into a particular patient. Our
goals allow for these factors to affect transplantation outcomes. For
example, current OPTN policies take into account the special medical
needs of children. The Secretary endorses this approach and expects
that the OPTN will continue to take these needs into account as it
develops new medical criteria and allocation policies.
Transition Protections (Sec. 121.8 (a)(5)) Finally, we have added a
requirement that transition protections (sometimes termed
``grandfather'' rights) be considered whenever a change in policy
disadvantages an identifiable set of patients already waiting on the
national list of transplant candidates.
To implement these protections, the OPTN would determine whether a
change disadvantaged some patients, and if so, consider developing a
transition policy to eliminate that disadvantage. The transition policy
would be submitted to the Department for review along with the new
policy, together with estimates of the likely effects of each. Because
a transition policy complicates organ allocation, and because the
Secretary wants to preserve OPTN flexibility to develop and implement
minor improvements with no consequential effect on existing patients'
priorities, the transition provision allows the OPTN some flexibility
as to whether, for how long, and for which patients the transition
procedure would be developed. Of course, the OPTN would be free to
devise particular approaches that would be most efficient and effective
for a particular patient population. As with all other allocation
policies, the Department would review each proposed transition
procedure.
In addition, the Secretary has adopted a special transition
provision for the first revision of the liver allocation policy. The
OPTN is directed to develop a transition proposal for the Secretary's
review which would, to the extent feasible, treat each individual on
the national list and awaiting transplantation on the date of the
publication of this regulation in the Federal Register no less
favorably than he or she would have been treated had the revised policy
not become effective. The transition procedures for this initial
revision of the liver allocation policy may be limited in duration or
applied only to individuals with greater than average medical urgency
if this would significantly improve administration of the list or if
such limitations would be applied only after accommodating a
substantial preponderance of those disadvantages by the change in the
policy. See Sec. 121.8(a)(5)(ii).
Kidneys pose potential problems because, unlike other organs, a
significant fraction of patients have already spent years on the
national list and turnover is much lower. On the other hand, transition
procedures may be particularly important for kidney patients for the
same reason. We request comments on the transition procedure generally
and specifically as to its suitability for kidney patients.
(a) Indicator Data (Sec. 121.8 (a)(4) and 121.8 (b)) In order to
assess how well the OPTN's current or proposed allocation policies
achieve the performance goals previously stated, the Secretary requires
the OPTN to collect and report indicator data on outcomes, and to
compare alternative policies against estimated or projected outcomes.
It is primarily against these indicators that the Secretary will
determine whether the OPTN's proposed revisions to organ allocation
policies will be approved. The Secretary expects the OPTN to develop
appropriate indicators, but has specified several of central concern.
These are: disparities in waiting times in status among transplant
programs (especially disparities among the sickest categories of
patient); life-years lost (both pre-and post-transplant); the number of
patients who die while waiting for a transplant, and the number of
patients mis-classified. Our requirements for performance indicators
are presented in Sec. 121.8(a)(4). See also, Sec. 121.8 (a)(3),
discussed earlier, for the allocation policies themselves.
Over the past year, a great deal of the debate and analysis of
alternative allocation policies has benefitted from the results of
computer-based modeling of liver allocation. While current modeling has
some limitations, it is nonetheless useful today and holds great
promise of assisting the OPTN in devising, as well as assessing,
policies. The Secretary expects the OPTN to develop and use such models
for all organs and to present results to the Department.
(b) Deadlines for Initial Reviews (Sec. 121.8(c)) The Secretary
expects the achievement of these goals to be an ongoing process as
medical technology, experience, and our understanding of
transplantation improve over time. Therefore, we have provided for
periodic policy revisions. However, for all organs other than livers,
the Secretary is requiring that the OPTN develop initial revised
policies to meet the goals, and to submit these within one year from
the effective date of this rule. For livers, the Secretary is requiring
development of policies that will meet these goals, to be submitted by
60 days from the effective date of this rule.
Shortly after this deadline the Secretary will take action with
respect to the OPTN liver allocation proposal, depending on the
information available to us as to which option best meets the
performance goals set out in this rule. During consideration, the
Secretary is committed to using a process allowing for effective
comment and presentation of alternatives. In order to minimize the time
needed to develop approved policies, the Secretary will follow
carefully the OPTN's progress in developing the new liver allocation
policies.
(c) Liver Allocation Policies The OPTN has wrestled with liver
allocation issues for a decade. A brief summary of this history helps
in understanding both the current OPTN policy and the Department's
approach in this regulation. One of the two main purposes of the
December hearing was to obtain additional information and views on
liver allocation.
UNOS adopted a liver allocation policy in 1986, the first year of
OPTN operations. The allocation policy featured a point system
assigning relative weights for medical urgency, blood group
compatibility and waiting
[[Page 16316]]
time to patients within distinct distribution units. This initial
system allocated organs first among all patients locally (with
``local'' waiting lists meaning the OPO procurement area, ranging from
a single transplant hospital's list to the combined lists of all
transplant hospitals in an entire State), then to patients in the OPTN
region. At the time this policy was adopted, the country was divided
into nine regions. Eventually, the number of regions was expanded to
the current eleven to reduce differences in population size among the
regions. Major differences still remain, however.
The liver allocation policy also included an informal emergency
voluntary sharing practice known as ``UNOS STAT'' whereby a transplant
hospital would notify the UNOS Organ Center (the 24-hour organ
placement operation maintained by UNOS) that a patient was critically
ill and expected to die within 24 hours without a transplant. The Organ
Center, in turn, would immediately notify all OPOs and transplant
programs of the urgent need. Should a liver become available, the OPO
could bypass the usual allocation process and the liver could be
directed to the UNOS STAT patient's hospital. In effect, UNOS STAT was
a system for sharing livers nationally, but only for the medically
neediest patients. Between 1987 and 1990, it is estimated that 15
percent of the patients who received transplants were designated as
UNOS STAT.
Objections were raised about the use of UNOS STAT, citing a lack of
formal, uniform rules governing its use, and a concern that it was
being used excessively or inappropriately. It was abolished by the OPTN
in 1991. In addition to eliminating the UNOS STAT category, the liver
allocation policy modified in 1991 expanded significantly the
definition of the most urgent category by redefining it to mean death
within seven days without a transplant (rather than 24 hours as in UNOS
STAT). The rationale for the change was to provide greater opportunity
within the formal allocation system for transplantation of chronically
ill patients as well as those with acute fulminant liver failure.
Waiting time accrual under the liver allocation criteria was also
modified to give greater priority to the most urgent patients. Status 1
(originally Status 4; in the discussion the sickest patients will
always be referred to as Status 1, the current definition) patients
were assigned the highest priority within the same distribution unit by
only allowing waiting time accrued by a patient while listed as Status
1 to count for liver allocation. The Status 1 criteria specified until
recently that such patients have a life expectancy of less than 7 days
without a liver transplant. Patients who are listed as Status 1
automatically revert to Status 2 after 7 days unless they are relisted
as Status 1 by an attending physician. Prior to this policy change, it
was possible for a patient who had been waiting a long time in a lower
status to accumulate enough waiting time points to give that patient
enough total points to be ranked higher than a patient who was a Status
1. The definitions of Status 2, 3, and 4 patients were, until changed,
as described below:
Status 2: Patients are continuously hospitalized in an acute care
bed for at least 5 days, or are in the intensive care unit. Continuous
hospitalization is required.
Status 3: Patients require continuous medical care but may be
followed at home or near the transplant hospital.
Status 4: Patients at home, functioning normally.
However, because the system allocates organs first locally, then
regionally or nationally only if no local patients are a good match for
the organ, and because at any time it is likely that the relatively few
(or no) local patients in Status 1 will match, many organs go to Status
2 and 3 patients despite their being ranked lower in medical priority.
In the mid 1990s, about two thirds of liver transplants were received
by patients waiting in the ``local'' area, about one fifth by patients
in the region and outside of the ``local'' area, and about one eighth
by patients outside the region. Therefore, the preference for ``local''
plays a significant role in determining a patient's likelihood of
receiving an organ. Under the current system, there is a wide range
among OPOs and the OPTN regions in the number of patients on the
waiting list, the number of donor livers available, and the ratio of
patients per donor. Consequently, patients in different locations have
disproportionate probabilities of being offered a liver under this
arrangement. Further, because fixed boundaries are used in local and
regional distribution, some patients nearest the site of the donor who
are otherwise highly ranked according to urgency or waiting time
continue to wait while less sick patients in the ``local'' region are
transplanted. As a result, some patients with higher medical urgency
die waiting for a liver while other patients with less medical urgency
receive a transplant.
Between 1990 and 1996, the number of liver transplant hospitals
performing at least one liver transplant increased from 75 to 110, and
the number of liver transplant programs performing 35 or more liver
transplants per year increased from 18 to 41. Liver transplants
increased from 2,676 to 4,012. Thus, patients have more transplant
hospitals from which to choose, but at the same time competition among
liver transplant programs for available livers has increased. During
1996, there were 8,026 registrations for a liver transplant.
Some people criticize this policy because livers are allocated
``local first'' to whomever is highest ranked in the local area of
procurement. Thus, less sick patients can be transplanted before sicker
patients in other local allocation areas. They believe that the sickest
patients should always be transplanted first regardless of their
location, because their lives are most at risk. In 1996, about 21
percent of liver patients transplanted were Status 1 and about 30
percent were Status 2. Almost 48 percent of transplanted patients were
Status 3, and less than 1 percent were Status 4.
The counter argument to this criticism is that, if sickest patients
are always given preference, there is a less efficient use of the
available livers, because the sickest patients (Status 1) have lower
survival rates than transplant recipients with other statuses. Others
say that if less sick patients receive lower preference than under the
current policy, more of them will become sicker while waiting and then
will have lower survival rates when they are eventually transplanted.
Optimally, patients should be transplanted at a time when they are sick
enough to benefit from a transplant, but not so sick that the risk of
losing the graft is heightened. OPTN data show, however, that at one
year after transplant there is about an 11 percentage point difference
in patient survival rates and 13 percentage point difference in graft
survival rates between former Status 1 and 2. Some argue that part of
this difference is due to a side effect of local preference rather than
greater risk of graft loss: Status 1 patients, they assert, often get
an inferior organ that was made available only after it was turned down
for use for any patient in another local procurement area.
Table 6, taken from pages 143 and 149 of the 1997 Annual Report of
the OPTN and Scientific Registry shows graft and patient survival rates
of liver transplant patients, by status:
[[Page 16317]]
Table 6.--Three Month and One Year Graft and Patient Survival Rates of Liver Transplant Patients by Status
----------------------------------------------------------------------------------------------------------------
3 Month survival rate One year survival rate
---------------------------------------------------
Waiting list status at transplant N Graft Patient Graft Patient
(percent) (percent) (percent) (percent)
----------------------------------------------------------------------------------------------------------------
Status 1....................................... 1,019 74.6 81.9 67.7 76.3
Status 2....................................... 1,562 84.0 89.8 77.1 83.6
Status 3....................................... 3,437 90.0 95.1 84.0 91.4
Status 4....................................... 91 87.8 97.6 82.2 93.7
Unknown........................................ 162 n.c. n.c. n.c. n.c.
Overall........................................ 6,271 85.4 91.6 79.1 87.0
----------------------------------------------------------------------------------------------------------------
Note: Covers patients transplanted 1994-95 for which a survival time could be determined.
n.c.=not calculated
Another frequent criticism of the current policy is that there is
wide variation in waiting times from one geographic area to another. A
counter argument is that this variation cannot be attributed entirely
to the allocation policy, because it may also be a function of patient
selection decisions and the number of organs procured locally. However,
the allocation policy, particularly as it relates to the size of the
initial allocation area, is a major determinant of variation in waiting
times. For livers, waiting time differentials among transplant
hospitals and among organ allocation areas vary by a factor of five or
more.
A third criticism of the ``local first'' policy is that it greatly
limits patient choice. If some non-local transplant hospitals do a
better job and attract more patients, these patients come to those
hospitals only at the price of a reduced chance for a transplant and
compete with each other for the limited supply of organs available
locally. A counter argument is that some patients prefer to list at
local hospitals and that an assured supply of local organs facilitates
this particular choice.
Consideration of Alternative Policies Following discussions with
the Department, which suggested that computer modeling be undertaken,
UNOS contracted with the Pritsker Corporation in 1995 to develop a
computer simulation model for liver allocation. The model presents the
hypothetical outcomes resulting from the application of a number of
alternative allocation policies. Among the many outcomes measured were:
patients transplanted, percentages of patients transplanted by status,
number of pre- and post-transplant deaths, median waiting times, and
distance from donor location to transplant location.
The Liver/Intestinal Transplantation Committee of the OPTN
considered seven policies that were most representative of all those
modeled, including a policy for national sharing proposed by the
University of Pittsburgh Medical Center (UPMC). The UPMC proposal and
the other options had also been modeled by the CONSAD Research
Corporation under contract with the UPMC. The Committee's subsequent
recommendations were reviewed by the OPTN Patient Affairs Committee and
by its Allocation Advisory Committee which put forth an alternate
proposal. This proposal included a modest component of regional sharing
of organs, but rejected major regional sharing as well as the national
sharing advocated by UPMC.
At its meeting in June 1996, the Board of Directors considered the
policies proposed by the Liver/Intestinal Committee and the Allocation
Advisory Committee, as well as the existing liver allocation policy.
The Board decided to change the existing policy in several ways,
including redefining Status 1 to include only patients with ``acute''
failure, placing other patients in intensive care into the broader
Status 2 group along with other patients of lesser urgency, eliminating
Status 4 as an urgency category for prioritizing liver transplant
candidates, and mandating regional rather than local sharing for the
newly defined Status 1 group (region for Status 1 allocation would be
the area encompassing the 20 percent of the total number of Status 1
and 2 candidates on the national list who are nearest to the available
organ). The Board of Directors then sent this proposal into an OPTN
public hearing process held in the fall. In November 1996, the Board
voted to adopt the new Status definitions, but to drop regional
sharing. This change was scheduled to take place in January 1997.
However, for the reasons described below, the Board suspended the new
Status definitions (except for dropping Status 4) and the previous
allocation system remained in place with little change.
At the Department's public hearing in December 1996, these system
revisions became a major issue. The de facto effect of the Board's
vote, as presented by many witnesses and uncontradicted by any
evidence, was substantially to disadvantage the group called ``chronic
crashers'', which had previously had a high priority as the predominant
group within Status 1. In effect, the Board had increased the priority
for ``acute'' patients with high medical urgency and little waiting
time at the expense of another group with almost equally high medical
urgency. While the Board did not present a formal rationale for the
change in the record of its meeting, the change appears to be premised
on the Board's belief that acute patients have a higher survival rate
if transplanted promptly, and were disadvantaged under the current
system, as well as its belief that some types of chronic liver disease,
for example liver disease caused by alcoholism (alcoholic liver disease
or ALD), had substantially lower survival rates.
As to the survival rate issue, the Department agrees with the
approach taken by the American Medical Association in its report that
supported the 1996 Code of Medical Ethics provisions discussed earlier.
The report noted, ``only very substantial differences in the likelihood
of benefit among patients are relevant to allocation decisions.'' In
fact, as reported in the UNOS Update magazine of September/October
1996, the ``acute'' category of fulminant liver failure actually has a
lower survival rate after transplant than most types of chronic liver
disease.
With respect to ALD, the Department notes that data presented at a
National Institutes of Health Workshop indicated, ``[r]ates of graft
and patient survival after liver transplant for ALD are excellent and
are similar to those for other chronic liver diseases. * * *''
As a result of the airing of these matters at the HHS hearing, the
OPTN Board of Directors rescinded its decision and placed the new
policy on hold (while allowing, however, limited
[[Page 16318]]
experimentation with broader sharing for ``acute'' patients in two OPTN
regions). The net effect was temporarily to restore the prior system.
At its meeting of June 25-26, 1997, the OPTN Board approved another
policy, which would favor ``acute'' over ``chronic crasher'' patients.
This revised policy puts the ``acute'' group first, the ``chronic
crasher'' group second, and less urgent patients lower. Whatever the
merits of giving preference to ``acute'' or ``chronic'' patients, these
changes do little to reduce the fundamental inequities affecting
patients across the country, the vast majority of whom have ``chronic''
liver disease. On the other hand, the new preference for ``acute''
patients exhibits a commendable understanding of the crucial argument
in favor of this group: medical urgency.
All of these policy priorities, ranging from STAT to ``acute'',
represent OPTN attempts to favor the most urgent needs. In its
performance goals, the Department retains and emphasizes this recurring
theme of OPTN policies regarding allocation of livers as well as other
organs.
In light of the extensive deliberative process within the OPTN, the
many policies that have been considered, the substantial technical
information available, the availability of two modeling tools that
provide approximate quantitative estimates of the differing effects of
alternative policies, and above all the demonstrated inequity of the
current liver allocation policies, the Department is not providing the
OPTN the same period of time to reform liver allocation policy that it
is providing for other organs. For all organs other than livers, the
OPTN has one year from the effective date of these regulations to
develop and submit to the Department allocation policies that meet the
aforementioned performance standards. For livers, the Secretary is
allowing 60 days from the effective date of these regulations. The
Secretary appreciates that this time is far shorter than normal OPTN
time frames, which include an opportunity for public comment. However,
lengthy deliberations have already occurred and a great deal of
information is available that will facilitate rapid reform. Moreover,
the regulation specifies that no further public comment need be
solicited by the OPTN before the deadline, although the OPTN may choose
to do so. Similarly, the OPTN may choose to begin this process
immediately if it believes that more time is required.
The final rule requires that the OPTN submit proposed transition
procedures at the same time that it submits the proposed new allocation
policy, together with supporting data. The Department will review these
materials expeditiously, along with alternative proposals and public
comments. The Department's plan is to obtain public input immediately
following the deadline for the OPTN proposal. Commenters may propose
alterations or alternatives. We ask that all proposals, whether from
the OPTN or commenters, identify likely effects on inequalities in
waiting times for patients of like medical urgency, on mortality, on
life-years, on likelihood of organ wastage, and on other outcomes of
importance.
The Secretary anticipates that similar procedures will be followed
for other organs. In assessing these reforms for both livers and other
organs, the Secretary will take into account that increased donation,
more objective listing standards, and objective medical criteria for
status categories all have significant potential for reducing
geographic inequities. However, the Secretary has seen no evidence
suggesting that fundamental inequities can be removed in the near
future without broader geographic sharing of organs.
This final rule has not established specific quantitative measures
that an OPTN liver allocation policy must attain to receive Secretarial
approval. We expect the OPTN to use its medical expertise and
consultative process to develop an appropriate policy. However, based
on the use of the performance goals as a regulatory framework, it is
unlikely that the Secretary would approve a policy that did not achieve
a significant reduction in the disparity of waiting times, particularly
for the most urgent patients.
(d) Directed Donation (Sec. 121.8(e)) Proposed Sec. 121.7(d) on
directed donation elicited several comments. Suggestions were made to
delete the section on the basis that it would be misconstrued, and to
refine it to take into account varying State laws. One commenter said
that it contradicts the intent of the National Organ Transplant Act,
and another said that directed donation should be discouraged but not
prohibited. The existing OPTN policy discourages directed donation to
designated groups or classes of people, but permits directed donation
to named individuals. This policy is consistent with provisions of the
Uniform Anatomical Gift Act, a model law that has been adopted by all
States. The Department has retained in the final rule the language of
proposed Sec. 121.7(d) permitting directed donation of organs to named
individuals. See, Sec. 121.8(e). It should be pointed out that the
final rule permits directed donation of an organ to named individuals
only.
8. Section 121.9--Designated Transplant Program Requirements
Section 1138 of the Social Security Act creates an extraordinarily
severe sanction for failure to comply with approved OPTN rules and
requirements. This, in turn, would make it unfair and impossible to
create standards higher than a threshold that any competent hospital
might attain. In the proposed rule, the Department suggested the idea
of ``designated transplant programs'' as a way around this dilemma.
Under this approach, failure to meet certain OPTN standards could
result in an inability to receive organs, without necessarily
jeopardizing either other transplant programs at the same institution
or all Medicare and Medicaid reimbursement. No commenters objected to
this approach, and no controversy over this approach surfaced at the
public hearing. Accordingly, the Department has decided to retain the
proposed approach, while improving it to reflect useful suggestions
from commenters.
Most of the commenters on this section of the proposed rule
recommended that the standards for the training and experience of
transplant surgeons and transplant physicians, required for designation
under proposed Sec. 121.8(a)(2), apply also to Medicare-approved
transplant programs designated under proposed Sec. 121.8(a)(1). Three
commenters suggested that transplant programs be designated on the
basis of a minimum volume of transplant procedures and on patient
survival standards, criteria now used in approving certain transplant
programs for reimbursement under Medicare. Another commenter said that
the NPRM was contradictory in admitting as OPTN members all Medicare-
approved transplant hospitals, while expressing concern about
proliferation of transplant hospitals and emphasizing that the
Department did not wish to exclude hospitals from entering the field of
transplantation. In the preamble to the proposed rule, the Department
stated that the criteria for designation under proposed
Sec. 121.8(a)(1) and (2) are complementary, providing designated
transplant program status to programs that meet Medicare standards, as
well as to non-Medicare-approved programs which meet other requirements
established by the OPTN. The Department's concern about the number of
transplant hospitals was expressed in the context of ``uncontrolled
proliferation of transplant facilities,'' that is, permitting
designated status
[[Page 16319]]
without a method of ensuring the quality of care. See 59 FR 46488.
The Department sees the merit in having uniform standards for
designated transplant programs, but believes that it would be
disruptive to impose them unilaterally at this time. Instead, the
Secretary will consider this issue in the context of revising the OPTN
and Medicare standards. In that light, the Department has asked the
OPTN contractor to consider developing standards regarding risk-
adjusted graft and patient survival rates, and possibly volume of
transplant procedures, if the latest scientific evidence supports such
standards. If appropriate, such standards could supplement the
requirements for designated transplant programs under Sec. 121.9,
following the notice and comment provisions of the Administrative
Procedure Act.
The OPTN contractor, UNOS, said that the OPTN would not be able to
provide patients with information about key personnel in Medicare-
approved transplant programs, because it would have such information
only for transplant programs designated under proposed
Sec. 121.8(a)(2). In addition, UNOS suggested that the OPTN be given
authority to collect, maintain, and distribute data on key personnel
for all transplant programs. The Department believes that the OPTN
should define such a role through its Board of Directors' policy
development process under Sec. 121.4, and has asked the contractor to
do so. Thus, explicit regulatory language is not required. In the
meantime, to the extent that information is not readily available from
the OPTN, we expect individuals to obtain it from the transplant
programs themselves.
Two commenters suggested that a conflict exists between proposed
Sec. 121.8(c) and proposed Sec. 121.3(d)(2) with respect to designation
of transplant programs and membership of transplant hospitals. Under
proposed Sec. 121.3(d)(2), the OPTN is directed to accept as members of
the OPTN transplant hospitals which meet the requirements of proposed
Sec. 121.3(c)(1) or (2). Under proposed Sec. 121.8(c), (now
Sec. 121.9(c)), the OPTN may accept or reject applications from
transplant programs for designated status. There is no conflict,
because membership under Sec. 121.3 does not confer designated status
under Sec. 121.9. One commenter said that proposed Sec. 121.8(a) should
indicate that designated transplant programs are also OPTN members. The
Department has edited that paragraph in accordance with the suggestion.
See, Sec. 121.9(a). We have also added to Sec. 121.9(c) a requirement
that the OPTN act ``within 90 days'' on requests for designated status,
making it comparable to the change made in Sec. 121.3(c)(3), discussed
above.
With respect to the disciplines listed in proposed
Sec. 121.8(a)(2)(v) as areas for collaborative involvement for
designated transplant programs, two commenters suggested adding
histocompatibility and immunogenetics. The Department has done so. See,
Sec. 121.9(a)(2)(v). The commenters also suggested that the term
``tissue typing'' in proposed Sec. 121.8(a)(2)(vi) be changed to
``histocompatibility testing.'' The change has been made. See,
Sec. 121.9(a)(2)(vi).
The Department also has added a provision at Sec. 121.9(a)(2)
requiring transplant programs to have adequate resources to provide
transplant services to their patients and promptly to notify the OPTN
and patients listed for transplantation if the program becomes
inactive. We are aware of at least one instance in which a transplant
program became inactive, yet did not advise its patients of its
inability to perform transplants. Such a situation also could lead to
use of the enforcement provisions of Sec. 121.10.
9. Section 121.10--Reviews, Evaluation, and Enforcement
Two comments were received on this section of the proposed rule. In
response to one comment, an editorial suggestion, the Department has
clarified proposed Sec. 121.9(b)(1)(iii) to indicate that compliance by
member OPOs and transplant hospitals with OPTN policies, as well as
regulations, is covered in reviews and evaluations carried out by the
OPTN. See, Sec. 121.10(b)(1)(iii).
The other comment was an expression of concern about patients
listed at transplant programs whose designated status to receive organs
for transplantation may be suspended. The Department wishes to assure
all who share this concern that the enforcement provisions of
Sec. 121.10(c) allow for an orderly phase-out and transition period
should such a situation occur. Under Sec. 121.10, the OPTN is required
to monitor the compliance of individual transplant programs, to report
to the Secretary the results of any reviews or evaluations that
indicate noncompliance, and to make recommendations for appropriate
action by the Secretary. The Secretary expects the OPTN to pay
particular attention to programs experiencing difficulty. The rule
further permits the Secretary to request more information from the OPTN
or from the alleged violator, or both, before accepting or rejecting
the OPTN's recommendations, or to take any other action the Secretary
deems necessary. We expect that enforcement of these provisions will
follow the pattern established by UNOS and member transplant hospitals
in seeking voluntary compliance with OPTN policies in the past. That
is, through a dialogue between the OPTN (and the Secretary, if
necessary) and the transplant hospital alleged to be in violation of
the rules, every effort will be made to reach a resolution before a
decision is made to suspend a transplant program's designated status.
It is the Secretary's intention that the OPTN develop a policy which
minimizes disruption and cost to patients, and keeps them informed. The
best interests of patient care will be paramount in monitoring and
enforcement of compliance with this rule. In this regard, we have also
elaborated on the procedures for OPTN reviews of transplant hospitals
and OPOs. The OPTN shall conduct those reviews in accordance with the
schedule specified by the Secretary and shall report progress on those
reviews to the Secretary. See Sec. 121.10 (b)(3) and Sec. 121.10(b)(4).
10. Proposed Section 121.10--Appeals of OPTN Policies and Procedures
The Department received two comments on this section of the
proposed rule. One commenter pointed out that appeals submitted to the
Secretary must be sufficiently clear and substantiated. We agree that
the Secretary must have appropriate information on which to base a
decision, and believe that the language of the proposed rule provides
the latitude needed for the Secretary to obtain such information. See,
Sec. 121.4(d). The other commenter expressed an opinion that the
Secretary's role in approving policies and deciding appeals could lead
to arbitrary and capricious actions, and suggested that the Secretary's
decisions be published in the Federal Register. Similar points were
raised in comments about proposed Secs. 121.3 and 121.7 regarding
publication of the Secretary's decisions on allocation and other
policies of the OPTN, discussed above.
The Secretary's authority under proposed Sec. 121.10(b) is not
dependent on appeal and may be exercised at any time. We have moved the
language of proposed Sec. 121.10(a) to Sec. 121.4(d). Because proposed
Sec. 121.10(b) is redundant in light of Sec. 121.4(b)(2) and (d), we
have deleted this section from the final rule.
[[Page 16320]]
11. Section 121.11--Record Maintenance and Reporting Requirements
Most of the comments on this section expressed concern that the
proposed rule falls short of needed protections of confidentiality, and
suggested as a model the protections delineated in MEDPAR, a Medicare
data system used by HCFA. We agree with the need to ensure protection
of confidentiality and believe that the protocols in MEDPAR may lend
themselves appropriately to the records falling within the purview of
Sec. 121.11. We also believe, however, that the design of a system to
protect the confidentiality of OPTN records should be left to the OPTN,
subject to the Secretary's review and the data release provisions of
this final rule. We expect the OPTN to submit for the Secretary's
consideration a policy which will protect the confidentiality of OPTN
records, but at the same time permit access by researchers to the OPTN
and Scientific Registry data bases. Thus, we have amended proposed
Sec. 121.11(a) to reflect that records must be maintained and made
available subject to policies of the OPTN and this final rule, as well
as to applicable limitations based on personal privacy. We have also
amended this section from the original proposal to clarify that the
OPTN must follow such standard practices as making its information
transactions and dissemination electronic to the extent feasible
(unless requested in hard copy), and in disseminating information to
include manuals and other explanatory materials as necessary to assure
that the material is easily and accurately understood and used. We have
also emphasized in Sec. 121.11(b) and elsewhere that the OPTN should
use rapidly advancing Internet technology to make information swiftly,
conveniently, and inexpensively available throughout the nation.
Two commenters suggested adding a requirement that member
transplant hospitals submit data to the Scientific Registry, a
repository of data on transplant recipients that is operated under
contract with the Department. Proposed Sec. 121.11(b)(1) requires that
the OPTN submit data to the Scientific Registry. We agree that a
parallel requirement for transplant hospitals and OPOs is also
appropriate, and have added it. See, Sec. 121.11(b)(2). Another
commenter suggested establishing a 90-day time limit for the submission
of data under proposed Sec. 121.11(b)(2). Such an explicit provision is
not necessary because proposed Sec. 121.11(b)(2) requires that
information be provided on a prescribed schedule. In addition, UNOS
suggested requiring the submission of cost data to the OPTN. Although
we believe the language of the proposed rule is broad enough to permit
the OPTN to request submission of such data, we have added to the final
rule the phrase ``and other information that the Secretary deems
appropriate.'' We have also corrected omissions in proposed
Sec. 121.11(b) by including the Secretary as a recipient of the
information. We have added to the reporting requirements the phrase
``the OPTN and the Scientific Registry as appropriate. . . .'' This
reflects the fact that some data which are to be reported or otherwise
made available to the public are held by the contractor operating the
Scientific Registry, while other data are held by the OPTN contractor.
The OPTN and the Scientific Registry are often asked by
researchers, payers, the press, patients, and others for data. We
appreciate the importance of the contractors' obligation to maintain
the confidentiality of patient-identified data. However, we also
recognize that data, collected as a consequence of Federally funded
contracts and of official designation as a contractor of the Federal
government, generally should be in the public domain. Even patient-
identified data can be shared with researchers who provide appropriate
protections against redisclosure. It is vitally important that bona
fide researchers and modelers have ready and timely access to detailed
data in order to explore ways to improve organ transplantation and
allocation. Therefore, information should be made available to the
public while protecting patient confidentiality. To correct the
oversight of omitting this activity from the proposed rule, we have
added Sec. 121.11(b)(1)(v) which requires the OPTN and the Scientific
Registry to respond promptly (normally within 30 days) and favorably to
requests from the public for data to be used for bona fide research or
analysis purposes, to the extent that the contractors' resources
permit, or as directed by the Secretary. The contractors may impose
reasonable charges for responding to such requests. Pursuant to Federal
government-wide policy under OMB Circular No. A-130, charges should
reflect only the marginal cost of preparing the data for dissemination,
not the cost of collecting or maintaining it.
We have also added language in paragraph Sec. 121.11(b)(1)(vi)
saying that the contractors must respond similarly to reasonable
requests from the public. The regulation does not require the
contractors to satisfy every request; however, the ability to charge
for data requests should enable the contractors to accommodate most
requests. In addition, the contractors would have to provide ready
access to data that it originally received from transplant hospitals
and OPOs, to these same institutions. See, Sec. 121.11(b)(1)(vii).
The Secretary has added language to Sec. 121.11(b)(2) making clear
that hospitals and OPOs must provide data directly to the Department
upon request, and must authorize the OPTN and Scientific Registry to
release data to the Department or others as provided in the regulation.
The OPTN has informed us of difficulties it has in complying with both
instructions from the Department and its perceived obligation to these
institutions not to disclose data that might be made public by the
Department. While we do not believe this to be a serious dilemma, we
have drafted the final rule to make it clear that any hospital or OPO
must, as a condition of its OPTN membership, make data available
without restriction for use by the OPTN, by the Scientific Registry, by
the Department, and in many circumstances by others, for evaluation,
research, patient information, and other important purposes. In this
regard, we particularly emphasize that we are requiring that current,
institution-specific performance data be made available so that
patients, payers, referring physicians, the press, and others can
appraise the quality of transplantation programs. The Congress made
this an obligation of the OPTN.
We have added language in Sec. 121.11(b)(1)(I)(B) stating that the
OPTN and the Scientific Registry shall submit to the Secretary
information the Secretary deems necessary to prepare the Report to
Congress required by section 376 of the Act, in order to clarify the
contractors' responsibility in this area.
To complete the articulation of this policy, we have added a new
paragraph (c) to Sec. 121.11, ``Public access to data.'' This paragraph
provides that the Secretary may release to the public information upon
determining that the release will serve the public interest. For
example, data on comparative costs and outcomes at different transplant
programs, information on waiting list time, and information on the
frequency with which transplant hospitals refuse offers of organs for
their listed patients, will assist patients and their families and
advisors in deciding where they wish to be transplanted. This release
of data is consistent with section 375 of the Act, 42 U.S.C. 274c,
which directs the Department to provide information to patients, their
families, and their physicians about transplantation resources and
about the comparative
[[Page 16321]]
costs and patient outcomes at each transplant hospital affiliated with
the OPTN, in particular. It is also consistent with the Department's
practice of having the contractor include in its published reports
extensive data, including transplant hospital-specific survival data.
The provisions of Sec. 121.11(c) were not included in the NPRM of
September 8, 1994. To delay the implementation of this paragraph would
be contrary to the public interest in that the decision-making of these
parties regarding this life-saving procedure should be fully informed
as soon as possible. The release of data is essential to allow
patients, their families, and their physicians to make the most
informed decisions possible about transplantation. Furthermore, the
release of these data is consistent with the above-cited section of law
and with the well-established practice of publishing center-specific
outcome data, and thus public comment prior to publication is
unnecessary.
The Secretary specifically requests comments on whether the above
provisions sufficiently achieve the several important purposes served
by provision of information to the OPTN, the Department, and the
public, while protecting patient privacy.
12. Section 121.12--Preemption
A new section regarding preemption has been added to the final
rule. This section does not require notice and comment rulemaking by
the agency, as it does not alter the rights and responsibilities of any
party. Instead, it simply applies the preemption principles derived
from the Supremacy Clause of the United States Constitution. The
Secretary is directed by section 372 to oversee a national system for
distribution of organs, and the policies of the OPTN currently require
organ sharing across State lines. The performance goals and indicators
articulated by these rules are almost certain to increase interstate
sharing.
At least one State has passed a law that appears to limit organ
sharing policies. A national organ sharing system based primarily on
medical need, with geographic considerations having less weight than at
present as an allocation criterion, would be thwarted if a State
required that, prior to sharing an organ with any other State, there be
a written agreement with that other State or a requirement that the
hospital or OPO first attempt to match the organ with an eligible
transplant candidate within the State, regardless of status.
Similarly, a State enforcing such a law would almost certainly
render impossible the compliance of transplant hospitals and OPOs
within that State with rules and requirements of the OPTN, and thus
would jeopardize their ability to obtain Medicare and Medicaid
reimbursement. This too would thwart the Federal scheme created by
Congress.
A further negative effect would flow from the enactment by
additional States of such restrictive laws. If more States were to
enact such laws, greater disruption in the allocation of organs under
the OPTN's policies would occur. Patients registered for transplants in
such States would almost certainly die as a result of the restrictions
on organ sharing, while other patients would receive organs even though
their transplants would not be approved until later under the OPTN's
policies. Accordingly, for policy as well as legal reasons, the
Department has added the preemption statement to the regulation.
The preceding discussion constitutes a Federalism Assessment, as
required by Executive Order 12612, and we certify that this rule was
assessed in light of the principles, criteria, and requirements of that
Order.
III. Economic and Regulatory Impact
A. Legal Requirements
A number of statutes and executive orders require us to analyze the
economic impacts of final rules.
Executive Order (E.O.) 12866 requires that all regulations reflect
consideration of alternatives, of costs, of benefits, of incentives, of
equity, and of available information. Regulations must meet certain
standards, such as avoiding unnecessary burden. Special analysis is
required for regulations which are ``significant'' because they create
economic effects of $100 million or more; create adverse effects on the
economy, public health, or other named categories; create serious
inconsistency with actions of another agency; or materially alter the
budgetary impact of entitlements and other programs or the rights and
obligations of recipients thereof; or raise novel legal or policy
issues.
The Regulatory Flexibility Act requires that we analyze regulations
to determine whether they create a significant impact on a substantial
number of small entities (for purposes of the Act, all not-for-profit
hospitals and all OPOs are categorized as small entities), and if so to
prepare a Regulatory Flexibility Analysis exploring ways to mitigate
adverse impact.
Executive Orders 12875 and 12612 (dealing, respectively, with
``Enhancing the Intergovernmental Partnership'' and ``Federalism'')
require that we review regulations to determine if they unduly burden
States, localities, or Indian tribes, or if they inappropriately
infringe upon the powers and responsibilities of States.
Section 202 of the Unfunded Mandates Reform Act of 1995 requires
that we determine whether regulations may result in the expenditure of
$100 million either by State, local, and tribal governments, or by the
private sector.
The Congressional review procedure of section 801(a)(2)(A) of title
5, United States Code, enacted in 1996, requires that rules with an
economic effect of $100 million or more or other comparable effects be
classified as ``major'', and that these rules may not take effect until
the Congress has had 60 days to review them.
We have determined that this rule will not have consequential
effects on States, local governments, or tribal governments, because it
affects primarily the operation of private sector OPTN functions and
the allocation of organs among patients based on their medical
condition. It will not require an expenditure of $100 million or more
by the private sector. Therefore, it does not meet the special
consultative requirements of the Unfunded Mandates Reform Act. We have
determined that it will not have a significant impact on a substantial
number of small entities, and so certify under the provisions of the
Regulatory Flexibility Act. However, because there is significant
concern over the effects of changes in allocation policies on smaller
hospitals, and because we considered as an alternative the possibility
of imposing quality standards on transplant hospitals, we have prepared
a voluntary Regulatory Flexibility Analysis (RFA). The analysis which
follows, together with the remainder of this preamble, constitutes an
RFA. We have also determined that this is an economically
``significant'' rule under E.O. 12866 and a ``major'' rule for purposes
of Congressional review of agency rulemaking. (This rule is also
``significant'' under E.O. 12866 because it ``materially alters'' the
rights of recipients--patients--of entitlement and grant programs). The
analysis that follows, together with the remainder of this preamble,
constitutes a Regulatory Impact Analysis (RIA) meeting these
requirements.
This combined Regulatory Impact Analysis and Regulatory Flexibility
Analysis also serves to analyze the effects of policies that we expect
to approve under the procedures put in place under this rule, and that
are assessed in this preamble, including all organ allocation policies
necessary to
[[Page 16322]]
implement the performance goals and indicators that we establish.
At the time of the proposed rule, we stated that it would be
premature to analyze alternatives because of the procedural emphasis of
the NPRM. We stated that we would analyze comparatively the range of
options that we considered, including the existing OPTN policies, based
on the comments and information we later received. Subsequent events
explained earlier in this preamble, and the information that we have
subsequently received, have made it both desirable and possible to
analyze qualitatively, and in part to quantify, the effects of the
substantive, non-procedural policies promulgated under this final rule.
We are far better able to quantify the effects of changes in liver
allocation policy than of changes in allocation policy for other
organs. However, we expect those changes to be qualitatively similar,
and this analysis covers all allocation policies.
B. Effects of Organ Transplantation
Industry Structure and Size. As indicated in Table 7 below,
covering selected organs, transplantation services are a very
substantial set of medical procedures, although only a very small
fraction of the trillion dollar health care sector.
Table 7.--Estimated Billed Charges for Transplants, 1996
----------------------------------------------------------------------------------------------------------------
Average Total Average
billed program program
No. trans- charges per billed billed
Major organ No. programs 1996 plants 1996 transplant charges charges
1996 1996 1996
($1000s) ($1000s) ($1000s)
----------------------------------------------------------------------------------------------------------------
Kidney.............................. 253................... 11,099 $94 $1,043,306 $4,124
Liver............................... 120................... 4,058 290 1,176,820 9,807
Pancreas............................ 120................... 1,022 110 112,420 937
Heart............................... 166................... 2,342 228 533,976 3,217
Lung................................ 94.................... 805 241 194,005 2,064
Total programs...................... 753................... 19,366 ........... 3,060,527 ...........
Total hospitals..................... 281................... 19,366 ........... 3,060,527 10,892
----------------------------------------------------------------------------------------------------------------
Sources: Data on numbers of programs and hospitals 1996 Annual Report of the OPTN, page 20 and C-2. Data on
transplants performed from Facts About Transplantation in the U.S., UNOS, July 23, 1997. Data on billed
charges per transplant from ``Cost Implications of Human Organ and Tissue Transplantations, an Update: 1996,''
by Richard H. Hauboldt, F.S.A., of Milliman & Robertson, page 30, excluding OPO charges.
These data show that on average, transplant programs generate
revenues in the millions of dollars. Since most transplant hospitals
operate several programs, the unduplicated revenue average across the
281 transplant hospitals that are OPTN members is about $11 million
annually. This includes not just the cost of the transplant procedure
itself, but also pre- and post-transplant charges such as time in the
hospital waiting for a transplant. Because the source of these data
uses billed rather than negotiated charges, actual receipts may be
somewhat lower than shown above.
The range of revenues is much broader than these averages convey
because the number of transplants performed varies so widely. Table 8
below, taken from OPTN and Scientific Registry data, shows the dozen
highest volume programs for liver transplants performed in 1995 and
1996. These dozen programs performed one fourth of all liver
transplants. Taken together, the two dozen lowest volume programs of
those that performed transplants in 1996 only performed about 80
transplants, 2 percent of the total. Among active liver programs, the
median program performed about 30 transplants, while the average was
about 36.
Table 8.--12 of the Highest Volume Liver Transplant Programs, 1995-1996
------------------------------------------------------------------------
Transplant program 1995 Volume 1996 Volume
------------------------------------------------------------------------
UCLA Hospital Center, Los Angeles, CA......... 230 245
Presbyterian-University Hospital, Pittsburgh,
PA........................................... 209 179
Mount Sinai Medical Center, New York, NY...... 209 180
Jackson Memorial Hospital, Miami, FL.......... 194 179
Baylor University Medical Center, Dallas, TX.. 140 118
University of Chicago Medical Center, Chicago,
IL........................................... 132 130
University of California, San Francisco, CA... 106 100
University of Nebraska Medical Center, Omaha,
NE........................................... 94 81
Rochester Methodist Hospital, Rochester, MN... 91 89
University of Alabama Hospital, Birmingham, AL 82 86
Shands Teaching Hospital & Clinics,
Gainesville, FL.............................. 81 102
University of Michigan Hospital, Ann Arbor, MI 78 59
-------------------------
Total................................... 1,646 1,548
------------------------------------------------------------------------
Source: 1997 Annual Report of the OPTN, pp. 391-396
Thus transplant volumes, and revenues, are highly skewed, with the
average much higher than the median.
The billing cost data in Table 7 focus primarily on hospitals, and
do not include procurement charges, which average approximately $24,000
per major organ in 1996, for a total of approximately one-half billion
dollars
[[Page 16323]]
per year in addition to the $3 billion spent at transplant hospitals.
Procurement charges are paid through organ procurement organizations.
OPOs are by law given local (in some cases state-wide or larger)
monopolies through a review and designation system administered
directly by the Federal government. Currently, there are 63 of them,
averaging some $8 million annually in revenues. Most of the revenues of
both transplant programs and OPOs are paid by Federally funded health
programs, primarily Medicare and Medicaid, but also Federal Employees
Health Benefits Program (FEHBP), CHAMPUS, the Uniformed Services and
the VA. In total, the government is by far the largest single payer for
transplantation.
Included in the data above, but not separately identified, are
laboratory costs. These can be very substantial, as a wide range of
condition-related tests are necessary to monitor patient urgency, and
both donors and recipients must have a broad range of laboratory tests.
The data above also include follow-up charges for one year, but not
subsequent follow-up charges for immunosuppressive therapy and other
costs. These average, according to Milliman & Robertson, about $7,000
for pancreas, $16,000 for kidneys, and between $21,000 and $29,000 for
the other major organs in 1996. Adjusted for survival, Milliman &
Roberts estimate the five-year cost of major organ transplants
including follow-up costs as follows: heart, $317,000; liver, $394,00;
kidney, $172,000; lung, $312,000; and pancreas, $149,000.
There are other sources of data on these categories of costs, each
using somewhat different estimating techniques. Their estimates are
generally comparable though sometimes lower. We note that such figures
do not generally estimate the marginal cost of transplantation, after
subtracting other costs that would be incurred if the patient did not
receive an organ. Marginal costs are much lower. In the case of
kidneys, a number of studies have estimated that transplantation costs
are more than offset by reductions in other medical costs such as
dialysis costs.
For purposes of the Regulatory Flexibility Act, an entity is
considered ``small'' if it has revenues below a certain size threshold,
or operates as a not-for-profit entity that is not dominant in its
field. For health care providers, such as hospitals, the threshold
amount is $5 million in annual revenues. Taking into account total
hospital revenues and not just transplant revenues, few or no
transplant hospitals fall below this threshold. However, the great
majority of these institutions are not-for-profit entities, and hence
qualify as ``small entities'' despite their substantial revenues.
Patient Effects. Table 9 below provides dramatic evidence of the
importance both of increasing organ donation and of reducing
unnecessary deaths while waiting for organs. Unlike growth in the
waiting list, which in part reflects factors such as earlier and more
aggressive listing, these data on deaths while waiting for organs
provide clear evidence of the need for transplantation.
Table 9.--Reported Deaths on the Waiting List 1988-1996
--------------------------------------------------------------------------------------------------------------------------------------------------------
Organ
Year --------------------------------------------------------------------------------------------------
1988 1989 1990 1991 1992 1993 1994 1995 1996
--------------------------------------------------------------------------------------------------------------------------------------------------------
Kidney............................................... 739 759 917 975 1052 1285 1361 1510 1814
Kidney-Pancreas...................................... 0 0 0 0 15 61 71 86 91
Pancreas............................................. 6 23 21 37 33 3 13 4 5
Liver................................................ 195 284 316 435 495 562 657 799 954
Heart................................................ 494 518 612 779 780 763 724 769 746
Heart-Lung........................................... 61 77 68 45 44 51 48 28 48
Lung................................................. 16 38 50 139 219 252 286 340 385
Intestine............................................ 0 0 0 0 0 3 15 19 22
Overall.............................................. 1,502 1,666 1,962 2,360 2,580 2,902 3,055 3,421 4,065
--------------------------------------------------------------------------------------------------------------------------------------------------------
Source: UNOS web site at http://www.UNOS.org/sta--dol.htm, data as of January 13, 1997.
The approximately 20,000 annual transplants of major organs fall
into two broad groups. More than half are kidneys. In the case of
kidneys, dialysis is an alternative to transplantation for extended
periods of time. Therefore, for most patients transplantation is not a
matter of immediate survival. Instead, the benefits of transplantation
fall largely (though not exclusively) in the domain of improved quality
of life. These improvements can be very substantial, as physical health
while on dialysis is significantly impaired, and dialysis imposes major
stresses and substantial inconveniences in carrying out normal
activities. In sum, dialysis sustains life but not well-being whereas a
transplant can and often does restore well-being. For other organs, a
transplant is in most cases a matter of survival. There are life-
prolonging technologies that work for some patients (e.g., left
ventricular assist devices for hearts) but for most awaiting extrarenal
organs, a transplant is literally essential to survival. Thus, in round
numbers the annual benefits of organ transplantation include about
eleven thousand lives vastly improved by kidney transplantation, and
another eight thousand lives both vastly improved and prolonged by
transplantation of other major organs.
It is common, in benefit cost analysis, to use a concept termed
``value of a statistical life'' to estimate in monetary terms the
benefits from lives saved. Estimates of this value can be derived from
information on the preferences of individuals for reduction in the risk
of death, and their willingness to pay for such reductions. In this
case, however, it is important to take into account two major factors
that reduce the usefulness of a statistical life as a measure: (a) most
organ transplant recipients are much older than average and hence gain
fewer years than would average beneficiaries of other life-saving
interventions, and (b) an organ transplant carries a substantial risk
of either the graft or the patient not surviving. For example,
according to historical data from the 1997 Annual Report of the OPTN
(page 23), only 62 percent of cadaveric kidney grafts survive 5 years,
and only 81 percent of these patients survive 5 years (patient survival
is substantially higher because dialysis is usually an option if the
organ fails). Five year patient survival rates for livers 72 percent,
for hearts 67 percent, and for lungs 43 percent. As each year passes,
additional patients die, though at lower rates than in the first year
or two. Survival rates
[[Page 16324]]
have improved in recent years, but the statistical expectation of
increased longevity and/or graft survival from a transplant is on the
order of a dozen years (a rough estimate since we do not yet know what
the long-term experience will become), not the 40 years (half a
lifetime) that underlies most estimates of statistical lives. Using the
more conservative concept of a ``statistical life-year'' saved, then,
the benefit from each year's cohort of approximately eight thousand
non-renal transplant recipients approximates one hundred thousand life
years. In a recent rule-making on tobacco, HHS estimated the value of a
statistical life-year at about $116,000 (see Federal Register of August
28, 1996, at page 44576). This was a conservative estimate that would
reasonably apply to organ transplantation (though a figure several
times as high could equally reasonably be used). Applying the
conservative $116,000 value to statistical life-years saved by non-
renal organ transplants, the social benefit from each annual cohort of
recipients is on the order of $12 billion. (Additional benefits could
be calculated for quality of life improvements for kidney recipients.)
Thus, whether one counts lives saved, life-years extended, or improved
quality of life, and whether or not expressed as dollars, the social
benefits of transplantation far exceed the admittedly expensive costs
of transplantation.
C. Effects of This Rule
This rule creates three major effects. First, it establishes terms
of public oversight and accountability for the entire organ
transplantation system, and the OPTN in particular. We believe that
this reform creates major public benefits in the categories of ``good
government,'' preserving public trust and confidence in organ
allocation, and assuring the rule of law. The Secretary does not
believe that such oversight creates any consequential costs. Its
benefits are substantial, but intangible. They may well lie primarily
in future problems avoided (e.g., reduction in organ donation if the
public were to lose confidence in the fairness of the OPTN in
allocating organs) rather than in specific current problems solved.
Second, this rule requires creation of a system of patient-oriented
information on transplant program performance. At present, the
fundamentals of such a system exist through the efforts of the OPTN.
The OPTN collects, validates, and analyzes a great deal of important
information. It publishes, in collaboration with this Department, a
Report of Center Specific Graft and Patient Survival Rates. This report
consists of 9 volumes and 3,200 pages, and contains valuable
information. However, from a patient perspective it is not up-to-date
or easy to use. The most recent version was the 1997 report, but the
data were current only up through April, 1994. The primary limitations
of the Report are that the survival rates are for patients transplanted
several years earlier and that there is no information regarding the
waiting list at individual transplant centers. We believe the data
should be more current. In addition, we believe center specific waiting
times and numbers and percentages of transplant center organ turndowns
of organs for non-medical reasons should be made available to the
patients. Finally, versions are needed that are easy to use for
patients, physicians, and families who wish to compare center
performance on any or all of these dimensions.
Third, this rule will improve equity by creating performance goals
against which the OPTN can reform current allocation policies. Such a
reform has important benefits--though benefits virtually impossible to
quantify--in their own right. We note that ``equity'' is an important
goal under Executive Order 12866. Unfortunately, improved equity is an
extraordinarily difficult concept to quantify. It is a goal and as it
is achieved, benefits accrue to members of society at large, to donor
families, to transplant candidates, and to transplant recipients. We do
have some measures of additional benefits arising in part from improved
equity, such as life-years saved, but these are a separate category of
benefit. We believe that a system that allocates organs to those most
in need in accordance with sound medical judgment, but with as little
regard to geography as reasonable, has profound benefits quite apart
from those that are life saving.
Table 10 below summarizes a number of measures of the effects of
alternative approaches to improved equity in organ allocation, for
livers. Comparable data are not readily available for other organs, and
for a number of reasons liver transplants are particularly susceptible
to improvement (hearts, for example, are already shared regionally and
kidney patients have dialysis options). However, these liver data
suggest the kinds of improvements that can be made for other organs.
Table 10.--Summary of Measures of Alternative Approaches to Liver Allocation
----------------------------------------------------------------------------------------------------------------
Allocation Inpatient
1996 Policy committee first National
----------------------------------------------------------------------------------------------------------------
Percent transplanted by hospitalization:
Inpatient............................................... 59% 73% 96% 97%
Outpatient.............................................. 41% 27% 4% 3%
Share of organs:
Local................................................... 78% 44% 38% 20%
Regional................................................ 18% 28% 31% 6%
National................................................ 4% 28% 31% 74%
Number transplants:
Initial................................................. 10,992 10,998 10,451 10,231
Repeat.................................................. 1,663 1,659 2,189 2,425
---------------------------------------------------
Total................................................. 12,655 12,657 12,640 12,656
Number on waiting list at end............................... 11,534 11,788 12,729 13,050
One year survival rate...................................... 80% 81% 76% 73%
Deaths:
Pre-transplant.......................................... 3,704 3,599 3,168 2,963
Post-transplant......................................... 2,539 2,555 2,967 3,144
---------------------------------------------------
Total................................................. 6,243 6,154 6,135 6,107
Life-years:
Pre-transplant.......................................... 26,600 27,193 29,443 29,915
[[Page 16325]]
Post-transplant......................................... 24,712 24,840 22,759 21,765
---------------------------------------------------
Total................................................. 51,312 52,033 52,202 51,680
----------------------------------------------------------------------------------------------------------------
Source: These estimates all come from modeling runs created by the Pritsker Corporation for the OPTN. Most of
those results were included in information provided at OPTN Board of Directors meetings. All data cover a
three year period, and are not annual estimates. Actual data for 1996 do not necessarily agree with these
modeling estimates, which apply to future years.
These data show, in broad outline, the effects of several
alternative policies for liver allocation. We emphasize that none of
the alternatives modeled included the effects of improved listing and
status standards, and for that and other reasons discussed below, these
results cannot be taken as precise predictions of the effects of
changes.
These data also omit a large number of alternative policies that
have been modeled, in the interest of economy of presentation. Of
particular interest are a set of policies that deal with a family of
options that have been termed ``time and distance weighted.'' This
family of options seeks to minimize transportation of organs while
achieving equity based on medical urgency and waiting time. In effect,
organs are transported long distances only when there is no alternative
for patients with high priority. Organs are kept locally when only very
small differences in patient benefit could be achieved by regional or
national transportation. Depending on the precise weights given to
medical status, waiting time, and distance, inequities due to waiting
time disparities can be greatly reduced. (See testimony of Dr. John P.
Roberts of the University of California, San Francisco, presented at
the public hearing and two letters from Dr. Roberts included as Exhibit
L in the Liver and Intestinal Organ Transplantation Committee Report
presented to the OPTN Board of Directors for its meeting on June 25,
1997) .
In Table 10, some of the most studied options are presented. These
options focus increasingly on broader geographical sharing, and on
greater reliance on medical urgency, from left to right. The first
column simply presents the predicted results of 1996 policy. The
``Allocation Committee'' column shows the results of an option reviewed
and subsequently rejected by the OPTN Board in 1996, that would have
allocated organs to Status 1 (most urgent) patients across regions
comprising 20 percent of the eligible hospitalized patients. Other
patients would have received either a slightly improved or no chance at
organs from out of the local area. Thus, this represents a very modest
change towards regional sharing from current policy. The third column,
``Inpatient First'', shows the results of an option that would have
allocated organs first nationally to hospitalized patients, and only
then to Status 3 patients. The ``National'' column shows the results of
an option proposed by the University of Pittsburgh Medical Center that
would have allocated organs by status, primarily on a national basis,
from most to least urgent (even the ``National'' proposal preserved a
substantial role for local allocation, by allocating first to a local
patient in Status 1, then nationally, then to a local patient in Status
2, then nationally, etc.).
One very striking result is that even a modest policy change can
very substantially change the kinds and places of patients receiving
organs. The Allocation Committee option decreases the share of livers
allocated to non-hospitalized patients (Status 3 and 4) from 41 percent
to 27 percent, and decreases the number of organs shared locally from
78 percent to 44 percent.
Taking the remainder of the rows in order, broader sharing has no
consequential effect on the number of transplants, but raises the
number of repeat transplants, thereby reducing the number of
individuals transplanted. This is a consequence of transplanting very
sick patients who are more likely to reject an organ graft after
transplantation. The number on the waiting list rises when organs go
first to more urgent patients. This is both a good and bad outcome--
longer waiting is ``bad'' but not if the alternative for other patients
is death. Survival rates decrease with a priority to the most urgent
because the most urgent patients tend to have more advanced disease and
additional co-morbidities (as discussed below, we do not believe that
current simulation results accurately measure likely survival rates).
However, as shown in the estimate of deaths, the net effect of these
changes is to reduce premature death, despite the decrease in survival
rates. Of importance is that the net total change in deaths masks a
very pronounced difference in direction for deaths pre-transplant
(which are substantially reduced), and deaths post-transplant (which in
the Pritsker model increase almost enough to offset pre-transplant
lives saved--but see discussion below of the CONSAD model). Life-years
exhibit a similar pattern to deaths, but are arguably a better measure
of real effects. Over a longer period of years, the total number of
people dying under all options will approach equality--but only if
there is no increase in transplant survival rates through medical
progress. But a life-year lived is never ``lost'' and represents an
unambiguous gain for the patients who benefit. Unfortunately, the post-
transplant life-years increase very little or decrease under broader
sharing (as estimated by Pritsker), whereas the years on the waiting
list, not dying but not well, increase dramatically.
As shown both in the Pritsker results and in the CONSAD results
presented below, no organ allocation gains are free. Taking as an
example deaths under a National policy, the Pritsker model estimates
that over a three year period some 700 fewer people would die pre-
transplant, and some 600 more people would die post-transplant. These
are changes of one-fifth or more in the number dying in each group.
Both costs and benefits are very high, thus reducing the net benefit
substantially.
The CONSAD model produces generally similar results, but shows a
distinct difference in the magnitude of deaths and life-years (as shown
in Table 11):
[[Page 16326]]
Table 11.--Numbers of Pre- and Post-Transplant Deaths and Life Years Under Alternative Liver Allocation Policies
----------------------------------------------------------------------------------------------------------------
Allocation Inpatient
1996 Policy committee first National
----------------------------------------------------------------------------------------------------------------
Deaths:
Pre-transplant.......................................... 4,571 4,394 4,060 4,216
Post-transplant......................................... 2,468 2,487 2,734 2,527
---------------------------------------------------
Total................................................. 7,039 6,881 6,794 6,743
Life-years:
Pre-transplant.......................................... 15,093 17,837 19,580 18,683
Post-transplant......................................... 38,107 38,096 35,537 36,465
---------------------------------------------------
Total................................................. 51,200 53,933 55,117 55,148
----------------------------------------------------------------------------------------------------------------
Source: CONSAD model run dated March 24, 1997.
As shown, under the CONSAD model the net life saving and life-year
saving effects of broader sharing are much more pronounced, as well as
more favorable to post-transplant experience. CONSAD shows National
allocation preventing a net of over 300 deaths and saving a net of
almost 4,000 life-years, in contrast to Pritsker's estimate of about
140 deaths and about 400 life-years (though 900 life-years for
Inpatient First). These are not small differences. Under the Pritsker
model, deaths would decrease, and life-years would rise, only about 2
percent from current levels under the most favorable result for broader
sharing. Under the CONSAD model, deaths would decrease about 4 percent
and life-years would rise about 8 percent. Realistically, in view of
the modeling issues discussed below, a 2 percent difference may
represent less than the possible error in the model, though an 8
percent difference is much more robust--if the model parameters and
assumptions are accurate. But even the CONSAD results indicate that
improved allocation policies have at best a limited potential to
improve outcomes. In contrast, improved organ donation represents an
unambiguous and potentially much larger gain.
There are known differences in model assumptions and approaches
that illustrate the strengths and weakness of both efforts. The
Pritsker model results ``throw away'' the first of the four years
modeled, to show more clearly the long-term rather than transitional
effect of change. In contrast, the CONSAD model cumulates the results
of years one, two, and three, rather than two, three, and four. Since
many life-years and deaths occur in the transition year, totals vary
for this reason. Second, the Pritsker model assumes that all transplant
programs operate at the same effectiveness as in the early 1990's, all
through the modeling years. The CONSAD model, in contrast, assumes a
slow but steady increase in transplant program performance and patient
survival. This assumption naturally results in fewer deaths and more
life-years gained in CONSAD runs, differentially in favor of those who
would otherwise die but could now expect to survive.
One difficulty shared by both models is that the OPTN has not
released current data on transplant outcomes. Thus, these modeling
results rely on data centering around 1990 and 1991 (including several
years before and after) rather than on the latest outcome data. Because
current graft and patient survival rates are known to be higher, this
makes certain outputs, particularly graft survival rates, deaths, and
life-years, inaccurate. CONSAD attempts to estimate recent progress,
but this is not a complete substitute for better baseline data.
Showing the importance of progress over time, UNOS data show that
between 1990 and 1995, one year patient survival for liver transplant
recipients increased from 83 to 87 percent.
Neither model completely captures a variety of real world nuances.
For example, under current policies survival rates for the sickest
patients who receive organs from outside their local area may be
influenced adversely by the sometimes lower quality of the organs they
receive that have been turned down elsewhere. But no hard data exist,
and neither model attempts to estimate such an effect. Neither model
attempts to deal with a hypothetical breakthrough in technology.
Neither model deals with the ``friction'' involved in transporting
organs over broader geographic areas (although they do produce
estimates of increased organ travel); both assume no wastage or reduced
graft survival results. None of these differences or commonalities
imply a fatal weakness in either or both of these models, but simply a
recognition that simulation modeling is by its very nature a partial
and incomplete attempt to predict results with any number of
assumptions potentially affecting outcomes.
From the Department's perspective, what is most important about
these modeling results is that despite the somewhat different interests
of their sponsors and the potential bias that might result, and the
infant efforts that they represent, these two independent efforts agree
almost completely on the qualitative effects to be expected from
changes in allocation policies, and substantially on the magnitudes
involved as well.
More complex to display are measures that capture likely effects of
improved policies on disparities in waiting times. As discussed earlier
in this preamble, program-specific, area-specific, and region-specific
results look very different, because aggregation masks disparities.
However, even regional differences are substantial. Table 12 below
follows shows the disparities under the 1996 policy, the Allocation
Committee (regional) proposal, the Inpatient First proposal, and the
National (local first, then national) proposal, as measured in average
days waiting for a liver transplant:
[[Page 16327]]
Table 12.-- Analysis Average Days Waiting for a Liver Transplant Under Alternative Liver Allocation Policies
----------------------------------------------------------------------------------------------------------------
Allocation Inpatient
OPTN region 1996 Policy committee first National
----------------------------------------------------------------------------------------------------------------
Region 1.................................................... 102 123 110 105
Region 2.................................................... 126 120 121 124
Region 3.................................................... 23 70 81 109
Region 4.................................................... 91 91 100 113
Region 5.................................................... 121 113 109 119
Region 6.................................................... 56 107 94 107
Region 7.................................................... 118 113 105 110
Region 8.................................................... 110 116 106 122
Region 9.................................................... 119 99 107 115
Region 10................................................... 88 92 93 110
Region 11................................................... 70 76 88 123
Standard Deviation.......................................... 32.24 17.93 11.55 6.81
----------------------------------------------------------------------------------------------------------------
Source: CONSAD model run dated March 24, 1997.
In this table, the standard deviation entry measures the extent to
which Regional averages differ. The standard deviation is a statistical
measuring tool. In this context, it means that under the current system
about two-thirds of the regions are within 32.24 days of the average
(both longer and shorter), and the remaining one-third are more than
that many days longer or shorter than the average. As these results
show, even modest geographic sharing based on a proxy for medical need
greatly reduces disparities in waiting time, from a standard deviation
of 32.24 days under current policy to as few as 6.81 days under a
national system of distribution. (Of course, as discussed previously,
current measures of waiting time disparities are weak because the lack
of listing standards does not create uniform, status-related measures
that would be truly fair as tie-breaking criteria.)
Another dimension of improved equity arises from reducing the role
of ethically irrelevant characteristics such as race or insurance
coverage in organ allocation. We already know, from prior studies, that
racial minorities--particularly African Americans--may not benefit to
the extent that their medical need warrants. In the final rule, as
noted previously, we have tasked the OPTN to develop policies to reduce
socio-economic inequities. No data from the modeling efforts or other
sources enable us to predict precise effects, even if the full
potential of such policies were clear. However, to the extent that
improved allocation policies reduce the ability of patients, payers, or
physicians to ``game'' the system, it will necessarily benefit the more
disadvantaged patients.
The performance goals created by this rule do not directly mandate
any of the allocation options just discussed. Instead, we require the
OPTN to craft new policies that achieve those goals. To the extent that
the modeling results capture our expectations, we expect those reformed
policies to show results much more similar to the rightmost two columns
in tables above than to the leftmost two columns. But neither precise
policy nor expected results have been modeled yet. And neither modeling
effort purports to measure directly equity, except insofar as reduced
disparities in waiting time in status capture this goal.
One final effect of the Department's overall initiative is
extremely important, though not attributable to this regulation.
Increases in organ donation are an unambiguous benefit. If, as seems
possible, the package of initiatives proposed by the Department could
increase organ donation by 20 percent or more, the benefits in lives
saved and life-years increased would both dwarf the estimates of these
effects as calculated by the simulation models. Increased donation
would also reduce waiting times. However, it would not necessarily
reduce disparities in waiting times. Only more equitable organ sharing
policies can directly reduce such disparities.
D. Alternatives Considered
Throughout this preamble, we have presented and analyzed
alternatives that the Department considered. Many of those selected
have an importance unrelated to regulatory impact as such, or have
little or no economic effect. There were, however, two broad strategic
options that we elected not to pursue at this time.
First, we could have required volume or performance standards for
transplant programs. The possibility of such standards was presented at
the public hearings, even though we had never proposed specific
standards for consideration. A great deal of research evidence exists
on differences among transplant programs in survival rates (the most
common measure), and on how volume correlates with those rates.
Nonetheless, we rejected that approach for a number of reasons. There
are a number of technical problems with such standards that could have
been overcome to varying degrees. For example, a volume standard would
require an exception for new programs during a transition period or it
would forever preclude new programs either in the many areas of the
country that do not have such programs, or to compete with established
programs where those now exist. More difficult to solve, a quality
standard would have to deal with the variance introduced by small
programs. For example, assuming a particular program had a ``true''
performance rate of 50 percent for a particular procedure, and
performed the first four procedures with two successes and two
failures, the fifth procedure would result either in a 60 percent or 40
percent cumulative rate, making it look very much better or worse than
its true performance. Two or three favorable or unfavorable results in
a row would not be statistically unusual. Lucky or unlucky runs that
would substantially affect potential error in apparent versus ``real''
results are likely in some low volume transplant programs. Further, the
need to ``case mix adjust'' adds significant complexity, and more
variance. Yet another problem arises because standards imply ``pass-
fail'' rates which do not necessarily push better programs to even
higher performance. And still another arises because a standard set
today may be obsolete a year from now as performance generally
improves. Not unimportantly, virtually the consensus view of the
testimony on this subject at our public hearings opposed volume and
even quality standards, and favored
[[Page 16328]]
more and better information. Using better information, patients and
physicians can and will reward better transplant programs by their
choices, and exert pressure on all hospitals to improve. For these and
other reasons, we elected to require instead improved information on
transplant program performance. We believe that better information can
equal or exceed the benefits of ``pass-fail'' standards without their
potentially arbitrary and disruptive effects.
Nothing in this volume/quality position related to minimum volume
is intended to discourage large payers and prudent purchasers from
setting their own standards. There is a big difference between a single
national standard that every program must meet or be terminated, and
elective payer standards. We encourage payers to explore and set such
standards, which can even focus on levels of excellence that could not
reasonably be set as nationally uniform minimum levels. We also expect
the OPTN to explore setting standards of excellence, and to continue
both research and modeling on such standards.
A second set of strategic options revolved around the possibility
of imposing directly, at this time, specific allocation standards
focusing on geographic equity. Such options would have the advantage of
reducing known inequities, and could rest substantially on the very
competent work already performed both by the OPTN itself and other
entities. For example, without any change in medical criteria, an
``inpatient first'' allocation policy could be introduced for liver
allocation. A ``time and distance weighted'' allocation policy, with
high weight given to health status, could also greatly improve equity
without increasing average travel times for donor livers as much as
other options (see Table 13).
Table 13--Estimated Average Miles Transported of Donated Livers Under
Alternative Liver Allocation Policies
------------------------------------------------------------------------
Average
Option for liver allocation distance
in miles
------------------------------------------------------------------------
1996 Policy................................................... 161
National Sharing.............................................. 1,072
Time and Distance Proposal.................................... 242
------------------------------------------------------------------------
Source: CONSAD Modeling run provided to Dr. John Roberts December 11,
1996. This particular Time and Distance Proposal gives only medium
weight to health status directly but substantial weight to waiting
time, which is correlated.
We have not adopted this family of options because we believe that
the performance goal approach we have crafted is likely to produce
superior results quickly and maintain its relevance as technology
changes. With the cooperation of the OPTN in bringing its expertise to
bear, there is no reason why policies better than any yet proposed
cannot be developed. In this regard, improved listing criteria and
medical status criteria will both reduce the need for broader sharing
and increase the professional trust and confidence needed to make that
sharing work. Not only can most transplant programs expect to gain as
many organs for their patients as they lose, but their own most urgent
cases will benefit.
A third option would have been to take no action at this time, as
urged by some. Under this option, we would defer absolutely to the
OPTN's judgment in the operation of the network. We rejected it for a
number of reasons. These include the demonstrated need for improvements
in the equitable allocation of organs, the Secretary's vital oversight
role, and the need for a system to carry out the Department's legal
obligations, including decisions on what binding standards will be used
to determine whether hospitals can participate in the Medicare and
Medicaid programs.
E. Effects on Transplant Programs
A great deal of fear and concern was evidenced at the public
hearing over effects on transplant programs, particularly smaller
programs, if broader sharing were to occur. Many witnesses feared the
possibility that patients would select, and organs follow to, the
largest programs (some of these witnesses asserted, and others denied,
that the largest programs had the best outcomes). The Department
believes that such fears are exaggerated, for many reasons. Perhaps
most important of these is that any such effects will depend on the
policies that the OPTN itself will devise. We expect that the OPTN can
identify policies that achieve equity and medical goals for patients
without harming medical care institutions.
In the discussion that follows, we note again that the majority of
transplant hospitals are ``small entities'' under the Regulatory
Flexibility Act simply by virtue of their non-profit status, and that
there is no known correlation of size of transplant program with size
of parent institution (beyond the fact that most small hospitals do not
conduct transplant programs at all).
For the most part, the smaller transplant programs already compete
directly with larger programs, even within the ``local first''
allocation schemes, or have the only program in their metropolitan
area. As shown selectively in Table 14 below (covering one-fourth of
the States in alphabetical order), and graphically on the map below,
the approximately 112 liver transplant programs active in 1995 were
concentrated in a far smaller number of cities. In fact, about a dozen
States had no liver transplantation program at all.
Table 14--Number of Small, Medium and Large Volume Liver Programs in Selected States
----------------------------------------------------------------------------------------------------------------
No. small No. medium No. large
State City (<12) (12-34)="" (35="">) Total
----------------------------------------------------------------------------------------------------------------
AL................. Birmingham............................. 0 0 1 1
AK................. None in Alaska......................... 0 0 0 0
AR................. None in Arkansas....................... 0 0 0 0
AZ................. Phoenix................................ 1 0 0 1
Tucson................................. 0 1 0 1
CA................. Los Angeles area....................... 1 2 2 5
Sacramento............................. 1 0 0 1
San Diego area......................... 0 2 0 2
San Francisco Bay area................. 0 0 3 3
CO................. Denver................................. 2 0 1 3
CT................. Hartford............................... 1 0 0 1
New Haven.............................. 0 1 0 1
DC................. Washington area........................ 1 0 1 2
FL................. Gainesville............................ 0 0 1 1
Miami.................................. 0 0 1 1
[[Page 16329]]
GA................. Atlanta................................ 1 0 1 2
HI................. Honolulu............................... 1 0 0 1
IL................. Chicago................................ 0 2 2 4
IN................. Indianapolis........................... 0 1 1 2
--------------------------------------------------------------------------------------------
Total.............. 17 Cities.............................. 9 9 14 32
----------------------------------------------------------------------------------------------------------------
Source: OPTN and Scientific Registry data supplied to the Department, through 1995, dated March 1, 1996.
These 13 States and 17 metropolitan areas contain 32 liver
transplant programs (the hundreds of remaining metropolitan areas,
smaller cities, and rural areas in these States have no local
transplant programs--their patients must travel). Of the nine small
(fewer than 12 transplants annually) programs, four have no local
competitors. These four have effective local monopolies for those
patients (undoubtedly a majority) who would prefer local
transplantation if given a choice. The five with competitors are
already surviving strong competition in their own health market. Thus,
with or without changes in allocation policy that favor broader
sharing, these transplant hospitals have substantial advantages or a
demonstrated capacity to withstand competition for patients.
The map below shows the pattern of choice for the entire nation,
grouping all transplant hospitals into small and medium (less than 35
transplants) or large (35 or more transplants). It shows that most
transplant hospitals already share cities or are located in closely
adjacent cities.
BILLING CODE 4160-15-P
[[Page 16330]]
[GRAPHIC] [TIFF OMITTED] TR02AP98.001
BILLING CODE 4160-15-C
[[Page 16331]]
Another potential concern arises from the fact that on average,
smaller transplant hospitals serve relatively less sick patients and
larger transplant hospitals tend to handle more hospitalized patients
(Status 1 and 2) (there are numerous exceptions to these average
tendencies). If nothing else changed but the relative ability of the
sickest patients to obtain organs, smaller transplant hospitals would
be expected to lose transplant volume. One of the modeling firms,
CONSAD, addressed this issue. As summarized in Table 15, its modeling
shows the following percentage shares of patients transplanted at
medium and large transplant hospitals under the alternative policies
modeled, assuming no behavioral responses by the programs.
Table 15
----------------------------------------------------------------------------------------------------------------
Allocation Inpatient
Liver transplants 1996 Policy committee first National
(percent) (percent) (percent) (percent)
----------------------------------------------------------------------------------------------------------------
Large programs (>35)........................................ 40 45 51 52
Medium programs (12-34)..................................... 37 34 30 30
Smaller programs (>12)...................................... 24 21 19 18
----------------------------------------------------------------------------------------------------------------
Source: CONSAD modeling run, dated March 24, 1997.
This result assumes that programs continue their current policies
as to which patients they tend to transplant, e.g., that smaller
transplant hospitals do not more aggressively seek to retain the
sickest patients. That seems extremely unlikely. Why would a program
that is worried about volume not change its practices to improve its
volume? But even in this ``worst'' case for smaller centers, they still
perform 18 percent of total liver transplantation, and the medium
programs still perform 30 percent of total liver transplantation. Far
more likely, ``threatened'' programs will strengthen their programs and
attract as many or more patients than they do at this time.
Finally, all of these computer simulations assume that the number
of available organs remains unchanged. We believe that improved use of
OPOs in identifying candidates for donation and in contacting families
of potential donors to request permission can alone significantly
improve organ supply. Data suggest that the Pennsylvania mandatory
referral program has increased by about 40 percent the number of organ
donors. The other actions that the Department will take can also have
significant effects in increasing donation. Thus, it is quite likely
that transplant programs of all sizes will see volume increases from
the entire package of reforms. Our expectation that on average
donations can be raised by about 20% over two years would allow all
centers to increase the number of patients they transplant.
In sum, nothing in the available data nor reasonable expectations
as to future business strategies by transplantation programs suggest
either that smaller transplant hospitals will be driven out of business
or that patients in cities served by smaller centers will be deprived
of local service. However, the Department will monitor and review OPTN
practices and policies as to their potential impacts on transplant
institutions.
IV. Paperwork Reduction Act of 1995
This final rule contains information collections which have been
approved by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 and assigned control number 0915-0184
with an expiration date June 30, 1998. In addition, there are reporting
and disclosure requirements that have not yet been approved (as noted
in the table). The title, description, and respondent description of
all information collections are shown below with an estimate of the
annual reporting and record keeping burden. Included in the estimate is
the time for reviewing instructions, searching existing data sources,
gathering and maintaining the data needed, and completing and reviewing
the collection of information.
Title: Organ Procurement and Transplantation Network.
Description: Information will be collected from transplant
hospitals, organ procurement organizations, and histocompatibility
laboratories for the purpose of matching donor organs with potential
recipients, monitoring compliance of member organizations with system
rules, conducting statistical analyses, and developing policies
relating to organ procurement and transplantation.
The practical utility of the data collection is further enhanced by
requirements that the OPTN must report a variety of data to the
Secretary, including data on performance by organ and status category,
including program-specific data, OPO specific data, data by program
size, and data aggregated by organ procurement area, OPTN region, the
nation as a whole, and other geographic areas (Sec. 121.8(a)(4)(iv)).
The OPTN must also transmit proposed allocation policies and
performance indicators which will be used to assess the likely effects
of policy changes and to ensure that the proposed policies are
consistent with these rules.
The OPTN and Scientific Registry must make available to the public
timely and accurate information the performance of transplant programs,
and must respond to requests from the public for data needed for bona
fide research or analysis purposes or to assess the performance of the
OPTN or Scientific Registry, to assess individual transplant programs,
or for other purposes (Sec. 121.11(b)(1)(C)).
The OPTN must provide to each member OPO and transplant hospital
the plans and procedures for reviewing applications and for monitoring
compliance with these rules and OPTN policies. The OPTN must also
report to the Secretary on OPOs and transplant hospitals that may not
be in compliance with these rules or OPTN policies, and on their
progress toward compliance.
The OPTN and Scientific Registry are required to maintain and
manage the information on candidates, donors, and recipients.
Description of Respondents: Non-profit institutions and small
organizations.
[[Page 16332]]
Estimated Annual Reporting and Record Keeping Burden
----------------------------------------------------------------------------------------------------------------
Annual
frequency Average Annual
Section Activity Annual No. of re- burden per burden
spondents response hours
----------------------------------------------------------------------------------------------------------------
121.3(c)(2)........................... OPTN membership 30 *** 1 40 1,200
application
requirements for
OPOs, hospitals,
histocompatibility
laboratories.
121.6(c) ** (Reporting)............... Submitting criteria 900 1 0.1 90
for organ accept.
121.6(c) ** (Disclosure).............. Sending criteria to 900 1 0.1 90
OPOs.
121.7(b)(4)........................... Reasons for refusal. 900 38 0.1 3,400
121.7(e) *............................ Transplant to 900 .5 0.1 42
prevent organ
wastage.
121.9(b).............................. Certification 10 *** 1 2.0 20
application
requirements for
transplant programs.
121.11(b)(2) *........................ Transplant candidate 900 33 0.1 3,000
registration.
121.11(b)(2) *........................ Donor registration.. 63 159 0.2 2,000
121.11(b)(2) *........................ Potential Recipient. 63 476 0.1 3,000
121.11(b)(2) *........................ Donor 56 143 0.1 800
Histocompitability.
121.11(b)(2) *........................ Transplant Recipient 56 321 0.1 1,800
Histocom..
121.11(b)(2) *........................ Transplant Recipient 900 23 0.25 5,250
Registration.
121.11(b)(2) *........................ Transplant Recipient 900 128 0.2 23,000
Follow-up.
-------------------------------------------------------------------------
Total........................... .................. 1,059 ........... ........... 43,692
----------------------------------------------------------------------------------------------------------------
* The data collection forms for these activities have been approved by the Office of Management and Budget under
the Paperwork Reduction Act (OMB No. 0915-0157).
** These requirements have been submitted for OMB approval. These requirements will not be effective until the
Department obtains OMB approval.
*** Current members of the OPTN and currently certified transplant programs will not have to re-apply for
membership and certification following promulgation of the new regulation. Only new applicants will be
required to apply, one time.
The final rules also require OPOs and transplant hospitals to
maintain records, as follows:
------------------------------------------------------------------------
Section Requirement
------------------------------------------------------------------------
121.7(b)(4)......................... Documentation of reason for
refusal.
121.7(c)(2)......................... Documentation of suitability
tests.
121.11(a)(2)........................ Maintain records on organ donors
and recipients.
------------------------------------------------------------------------
According to staff of OPOs and transplant hospitals, such record
keeping is integral to the operation of these facilities. Therefore,
these record keeping requirements impose no additional burden. In
compliance with the requirement for opportunity for public comment on
proposed data collection projects (section 3506(c)(2)(A) of Title 44,
United States Code, as amended by the Paperwork Reduction Act of 1995,
Public Law 104-13), comments are invited on: (a) whether the proposed
collection of information is necessary for the proper performance of
the functions of the agency, including whether the information shall
have practical utility; (b) the accuracy of the agency's estimate of
the burden of the proposed collection of information; (c) ways to
enhance the quality, utility, and clarity of the information to be
collected; and (d) ways to minimize the burden of the collection of
information on respondents, including the use of automated collection
techniques or other forms of information technology.
A separate announcement will be published in the Federal Register
when the Department obtains Office of Management and Budget approval
for Sec. 121.6(c), which contains information collection requirements.
Written comments and recommendations concerning the proposed
information collection should be sent to: Patricia Royston, HRSA
Reports Clearance Officer, Room 14-36, Parklawn Building, 5600 Fishers
Lane, Rockville, MD, 20857. Comments should be received within 60 days
after publication of this document in the Federal Register.
List of Subjects in 42 CFR Part 121
Organ transplantation, Hospitals.
Dated: March 20, 1998.
Claude Earl Fox,
Acting Administrator, Health Resources and Services Administration.
Approved:
Donna E. Shalala,
Secretary.
Regulation Text
Accordingly, 42 CFR part 121 is added to subchapter K to read as
follows:
PART 121--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK
Sec.
121.1 Applicability.
121.2 Definitions.
121.3 The OPTN.
121.4 OPTN Policies; Secretarial Review and Appeals.
121.5 Listing requirements.
121.6 Organ procurement.
121.7 Identification of organ recipient.
121.8 Allocation of organs.
121.9 Designated transplant program requirements.
121.10 Reviews, evaluation, and enforcement.
121.11 Record maintenance and reporting requirements.
121.12 Preemption.
Authority: Sections 215, 371-376 of the Public Health Service
Act (42 U.S.C. 216, 273-274d); Sections 1102, 1106, 1138 and 1872 of
the Social Security Act (42 U.S.C. 1302, 1306, 1320b-8 and 1395ii).
Sec. 121.1 Applicability.
(a) The provisions of this part apply to the operation of the Organ
Procurement and Transplantation Network (OPTN) and to the Scientific
Registry.
(b) In accordance with Section 1138 of the Social Security Act,
hospitals in which organ transplants are performed and which
participate in the programs under titles XVIII or XIX of that Act, and
organ procurement organizations designated under Section 1138(b)(1)(F)
of the Social Security Act, are subject to the requirements of this
part.
Sec. 121.2 Definitions.
As used in this part--
Act means the Public Health Service Act, as amended.
Designated transplant program means a transplant program that has
been
[[Page 16333]]
found to meet the requirements of Sec. 121.9.
Family member means a family member of a transplant candidate,
transplant recipient, or organ donor.
National list means the OPTN computer-based list of transplant
candidates nationwide.
OPTN computer match program means a set of computer-based
instructions which compares data on a cadaveric organ donor with data
on transplant candidates on the national list and ranks the candidates
according to OPTN policies to determine the priority for allocating the
donor organ(s).
Organ means a human kidney, liver, heart, lung, or pancreas, and
for purposes of the Scientific Registry, the term also includes bone
marrow.
Organ donor means a human being who is the source of an organ for
transplantation into another human being.
Organ procurement organization or OPO means an entity so designated
by the Secretary under Section 1138(b) of the Social Security Act.
Organ procurement and transplantation network or OPTN means the
network established pursuant to Section 372 of the Act.
Potential transplant recipient or potential recipient means a
transplant candidate who has been ranked by the OPTN computer match
program as the person to whom an organ from a specific cadaveric organ
donor is to be offered.
Scientific Registry means the registry of information on transplant
recipients established pursuant to Section 373 of the Act.
Secretary means the Secretary of Health and Human Services and any
official of the Department of Health and Human Services to whom the
authority involved has been delegated.
Transplant candidate means an individual who has been identified as
medically suited to benefit from an organ transplant and has been
placed on the national list by the individual's transplant program.
Transplant hospital means a hospital in which organ transplants are
performed.
Transplant physician means a physician who provides non-surgical
care and treatment to transplant patients before and after transplant.
Transplant program means a component within a transplant hospital
which provides transplantation of a particular type of organ.
Transplant recipient means a person who has received an organ
transplant.
Transplant surgeon means a physician who provides surgical care and
treatment to transplant recipients.
Sec. 121.3 The OPTN.
(a) Composition of the Board. (1) The OPTN shall establish a Board
of Directors of whatever size the OPTN determines appropriate, provided
that it includes at least the following members:
(i) Six members representing the following categories (two members
from each category):
(A) Transplant coordinators;
(B) Organ procurement organizations;
(C) Histocompatibility experts;
(ii) Eight individuals representing transplant candidates,
transplant recipients, organ donors, and family members;
(iii) Ten members from the following categories (two members each):
(A) Transplant surgeons;
(B) Transplant physicians;
(C) Transplant hospitals;
(D) Voluntary health associations; and
(E) Other experts from related fields including medical examiners,
hospital administration, or donor hospital personnel in such fields as
trauma, emergency medical services, critical care, neurology, or
neurosurgery; and
(iv) Six members from the general public from fields such as
behavioral science, computer science, economics, ethics, health care
financing, law, policy analysis, sociology, statistics, or theology.
These members need not have technical expertise in organ donation or
allocation.
(2) None of the members who are transplant recipients, transplant
candidates, organ donors, family members, or general public members
under paragraph (a)(1) of this section shall be employees of, or have a
similar relationship with, the categories of members listed in
paragraph (a)(1)(i) or paragraph (a)(1)(iii) or the OPTN.
(3) The Board of Directors shall include:
(i) Individuals representing the diversity of the population of
transplant candidates and recipients served by the OPTN, including, to
the extent practicable, minority and gender representation reflecting
the population of potential transplant candidates served by the OPTN;
(ii) No more than 50 percent transplant surgeons or transplant
physicians; and
(iii) At least 25 percent transplant candidates, transplant
recipients, organ donors and family members.
(4) Individuals on the Board shall be elected for a two-year term.
(b) Duties of the OPTN Board of Directors. (1) Executive Committee.
The Board of Directors shall elect an Executive Committee from the
membership of the Board. The Executive Committee shall include at least
one member who is a transplant candidate, transplant recipient, organ
donor, or family member; one general public member, one OPO
representative, and not more than 50 percent transplant surgeons and
transplant physicians.
(2) Executive Director. The Board of Directors shall appoint an
Executive Director of the OPTN. The Executive Director may be
reappointed upon the Board's determination that the responsibilities of
this position have been accomplished successfully.
(3) Committees. The Board of Directors shall establish such other
committees as are necessary to perform the duties of the OPTN.
Committees established by the Board of Directors shall include:
(i) Representation by transplant coordinators, organ procurement
organizations, and transplant hospitals, and at least one transplant
candidate, transplant recipient, organ donor or family member; and
(ii) To the extent practicable, minority and gender representation
reflecting the diversity of the population of potential transplant
candidates served by the OPTN.
(4) The Board of Directors shall develop and propose policies for
the equitable allocation of organs, as described in Sec. 121.8.
(c) Membership of the OPTN. (1) The OPTN shall admit and retain as
members the following:
(i) All organ procurement organizations;
(ii) Transplant hospitals participating in the Medicare or Medicaid
programs; and
(iii) Other organizations, institutions, and individuals that have
an interest in the fields of organ donation or transplantation.
(2) To apply for membership in the OPTN:
(i) An OPO shall provide to the OPTN the name and address of the
OPO, and the latest year of designation under section 1138(b) of the
Social Security Act;
(ii) A transplant hospital shall provide to the OPTN the name and
address of the hospital, a list of its transplant programs by type of
organ; and
(iii) Any other organization, institution, or individual eligible
under paragraph (c)(1)(iii) of this section shall demonstrate to the
OPTN an interest in the fields of organ donation or transplantation.
(3) The OPTN shall accept or reject as members entities or
individuals
[[Page 16334]]
described in paragraph (c)(1)(iii) of this section within 90 days.
(4) Applicants rejected for membership in the OPTN may appeal to
the Secretary. Appeals shall be submitted in writing within 30 days of
rejection of the application. The Secretary may:
(i) Deny the appeal; or
(ii) Direct the OPTN to take action consistent with the Secretary's
response to the appeal.
(d) Corporate Status of the OPTN. (1) The OPTN shall be a private,
not-for-profit entity.
(2) The requirements of this section do not apply to any parent,
sponsoring, or affiliated organization of the OPTN, or to any
activities of the contracting organization that are not integral to the
operation of the OPTN. Such an organization is free to establish its
own corporate procedures.
(3) No OPTN member is required to become a member of any
organization that is a parent, sponsor, contractor, or affiliated
organization of the OPTN, to comply with the by-laws of any such
organization, or to assume any corporate duties or obligations of any
such organization.
(e) Effective date. The organization designated by the Secretary as
the OPTN shall have six months from July 1, 1998, or six months from
its initial designation as the OPTN, whichever is later, to meet the
board composition requirements of paragraph (a) of this section. The
organization designated by the Secretary as the OPTN shall have six
months from July 1, 1998, or six months from initial designation as the
OPTN, whichever is later, to meet any other requirements of this
section, except that the Secretary may extend such period for good
cause.
Sec. 121.4 OPTN policies: Secretarial review and appeals.
(a) The OPTN Board of Directors shall be responsible for
developing, with the advice of the OPTN membership and other interested
parties, policies within the mission of the OPTN as set forth in
section 372 of the Act and the Secretary's contract for the operation
of the OPTN, including:
(1) Policies for the equitable allocation of cadaveric organs in
accordance with Sec. 121.8;
(2) Policies, consistent with recommendations of the Centers for
Disease Control and Prevention, for the testing of organ donors and
follow-up of transplant recipients to prevent the spread of infectious
diseases;
(3) Policies that reduce inequities resulting from socioeconomic
status, including, but not limited to:
(i) Ensuring that patients in need of a transplant are listed
without regard to ability to pay or source of payment;
(ii) Procedures for transplant hospitals to make reasonable efforts
to make available from their own resources, or obtain from other
sources, financial resources for patients unable to pay such that these
patients have an opportunity to obtain a transplant and necessary
follow-up care;
(iii) Recommendations to private and public payers and service
providers on ways to improve coverage of organ transplantation and
necessary follow-up care; and
(iv) Reform of allocation policies based on assessment of their
cumulative effect on socioeconomic inequities;
(4) Policies regarding the training and experience of transplant
surgeons and transplant physicians in designated transplant programs as
required by Sec. 121.9;
(5) Policies for nominating officers and members of the Board of
Directors; and
(6) Policies on such other matters as the Secretary directs.
(b) The Board of Directors shall:
(1) Provide opportunity for the OPTN membership and other
interested parties to comment on proposed policies and shall take into
account the comments received in developing and adopting policies for
implementation by the OPTN; and
(2) Provide, at least 30 days prior to their proposed
implementation, proposed policies to the Secretary, who may provide
comments and/or objections within a reasonable time, or may publish the
policies in the Federal Register to obtain comments from the public.
The Board of Directors shall indicate which of the proposed policies it
recommends be enforceable under Sec. 121.10. If the Secretary seeks
public comments, these comments will be considered and may affect
subsequent response to the OPTN. The OPTN shall take into account any
comments the Secretary may provide. If the Secretary objects to a
policy, the OPTN may be directed to revise the policy consistent with
the Secretary's direction. If the OPTN does not revise the policy in a
timely manner or if the Secretary otherwise disagrees with its content,
the Secretary may take such other action as the Secretary determines
appropriate.
(c) The OPTN Board of Directors shall provide the membership and
the Secretary with copies of the policies as they are adopted, and make
them available to the public upon request. The Secretary will publish
lists of these documents in the Federal Register, indicating which ones
are subject to the special compliance requirements and potential
sanctions of section 1138 of the Social Security Act. The OPTN shall
also continuously maintain OPTN policies for public access on the
Internet, including current and proposed policies.
(d) The OPTN, or its members, or other individuals, or entities
objecting to policies developed by the OPTN or the Secretary may submit
appeals to the Secretary in writing. Any such appeal shall include a
statement of the basis for the appeal. The Secretary will seek the
comments of the OPTN on the issues raised in the appeal of an OPTN-
developed policy. Policies remain in effect during the appeal. The
Secretary may:
(1) Deny the appeal;
(2) Direct the OPTN to revise the policies consistent with the
Secretary's response to the appeal, or
(3) Take such other action as the Secretary determines appropriate.
(e) The OPTN shall implement policies and:
(1) Provide information to OPTN members about these policies and
the rationale for them.
(2) Update policies developed in accordance with this section to
accommodate scientific and technological advances.
Sec. 121.5 Listing requirements.
(a) A transplant hospital which is an OPTN member may list
individuals only for a designated transplant program.
(b) Transplant hospitals shall assure that individuals are placed
on the national list as soon as they are determined to be candidates
for transplantation. The OPTN shall advise transplant hospitals of the
information needed for such listing.
(c) An OPTN member shall pay a registration fee to the OPTN for
each transplant candidate it places on the national list. The amount of
such fee shall be determined by the OPTN with the approval of the
Secretary. No less often than annually, and whether or not a change is
proposed, the OPTN shall submit to the Secretary a statement of its
proposed registration fee, together with such supporting information as
the Secretary finds necessary to determine the reasonableness or
adequacy of the fee schedule and projected revenues. This submission is
due at least three months before the beginning of the OPTN's fiscal
year. The Secretary will approve, modify, or disapprove the amount of
the fee within a reasonable time of receiving the OPTN's submission.
[[Page 16335]]
Sec. 121.6 Organ procurement.
The suitability of organs donated for transplantation shall be
determined as follows:
(a) Tests. An OPTN member procuring an organ shall assure that
laboratory tests and clinical examinations of potential organ donors
are performed to determine any contraindications for donor acceptance,
in accordance with policies established by the OPTN.
(b) HIV. Organs from individuals known to be infected with human
immunodeficiency virus shall not be procured for transplantation.
(c) Acceptance criteria. Transplant programs shall establish
criteria for organ acceptance, and shall provide such criteria to the
OPTN and the OPOs with which they are affiliated.
Sec. 121.7 Identification of organ recipient.
(a) List of potential transplant recipients. (1) An OPTN member
procuring an organ shall operate the OPTN computer match program within
such time as the OPTN may prescribe to identify and rank potential
recipients for each cadaveric organ procured.
(2) The rank order of potential recipients shall be determined for
each cadaveric organ using the organ specific allocation criteria
established in accordance with Sec. 121.8.
(3) When a donor or donor organ does not meet a transplant
program's donor acceptance criteria, as established under
Sec. 121.6(c), transplant candidates of that program shall not be
ranked among potential recipients of that organ and shall not appear on
a roster of potential recipients of that organ.
(b) Offer of organ for potential recipients. (1) Organs shall be
offered for potential recipients in accordance with policies developed
under Sec. 121.8 and implemented under Sec. 121.4.
(2) Organs may be offered only to potential recipients listed with
transplant programs having designated transplant programs of the same
type as the organ procured.
(3) An organ offer is made when all information necessary to
determine whether to transplant the organ into the potential recipient
has been given to the transplant hospital.
(4) A transplant program shall either accept or refuse the offered
organ for the designated potential recipient within such time as the
OPTN may prescribe. A transplant program shall document and provide to
the OPO and to the OPTN the reasons for refusal and shall maintain this
document for one year.
(c) Transportation of organ to potential recipient. (1)
Transportation. The OPTN member that procures a donated organ shall
arrange for transportation of the organ to the transplant hospital.
(2) Documentation. The OPTN member that is transporting an organ
shall assure that it is accompanied by written documentation of
activities conducted to determine the suitability of the organ donor
and shall maintain this document for one year.
(3) Packaging. The OPTN member that is transporting an organ shall
assure that it is packaged in a manner that is designed to maintain the
viability of the organ.
(d) Receipt of an organ. Upon receipt of an organ, the transplant
hospital responsible for the potential recipient's care shall determine
whether to proceed with the transplant. In the event that an organ is
not transplanted into the potential recipient, the OPO which has a
written agreement with the transplant hospital must offer the organ for
another potential recipient in accordance with paragraph (b) of this
section.
(e) Wastage. Nothing in this section shall prohibit a transplant
program from transplanting an organ into any medically suitable
candidate if to do otherwise would result in the organ not being used
for transplantation. The transplant program shall notify the OPTN and
the OPO which made the organ offer of the circumstances justifying each
such action within such time as the OPTN may prescribe.
Sec. 121.8 Allocation of organs.
(a) Policy development. The Board of Directors established under
Sec. 121.3 shall develop, in accordance with the policy development
process under Sec. 121.4, organ-specific policies (including
combinations of organs, such as for heart-lung transplants) for the
equitable allocation of cadaveric organs among potential recipients.
Such policies shall meet the requirements in paragraphs (a)(1), (2),
(3), (4) and (5) of this section. Such policies shall be reviewed
periodically and revised as appropriate.
(1) Minimum listing criteria for including transplant candidates on
the national list shall be standardized and, to the extent possible,
shall contain explicit thresholds for listing a patient and be
expressed through objective and measurable medical criteria.
(2) Transplant candidates shall be grouped by status categories
ordered from most to least medically urgent, with a sufficient number
of categories to avoid grouping together persons with substantially
different medical urgency. Criteria for status designations shall
contain explicit thresholds for differentiating among patients and
shall be expressed, to the extent possible, through objective and
measurable medical criteria.
(3) Organ allocation policies and procedures shall be in accordance
with sound medical judgment and shall be designed and implemented:
(i) To allocate organs among transplant candidates in order of
decreasing medical urgency status, with waiting time in status used to
break ties within status groups. Neither place of residence nor place
of listing shall be a major determinant of access to a transplant. For
each status category, inter-transplant program variance in the
performance indicator ``waiting time in status'' shall be as small as
can reasonably be achieved, consistent with paragraph (a)(3)(ii) of
this section. Priority shall be given to reducing the waiting time
variance in the most medically urgent status categories before reducing
the waiting time variance in less urgent status categories, if
equivalent reductions cannot be achieved in all status categories; and
(ii) To avoid futile transplantation, to avoid wasting organs, and
to promote efficient management of organ placement.
(4) The OPTN shall:
(i) Develop mechanisms to promote and review compliance with each
of these goals;
(ii) Develop performance indicators to facilitate assessment of how
well current and proposed policies will accomplish these goals;
(iii) Use performance indicators, including indicators described in
paragraph (a)(4)(iv) of this section, to establish baseline data on how
closely the results of current policies approach these goals and to
establish the projected amount of improvement to result from proposed
policies; and
(iv) Timely report data to the Secretary on performance by organ
and status category, including program-specific data, OPO specific
data, data by program size, and data aggregated by organ procurement
area, OPTN region, the nation as a whole, and such other geographic
areas as the Secretary may designate. Such data shall include inter-
transplant program variation in waiting time in status, total life
years pre- and post-transplant, patient and graft survival rates
following transplantation, patients mis-classified by status, and
number of patients who die waiting for a transplant. Such data shall
cover such intervals of time, and be presented using confidence
intervals or other measures of variance, as appropriate to avoid
spurious results or erroneous interpretation due to small numbers of
patients covered.
[[Page 16336]]
(5) Transition. (i) General. When the OPTN revises organ allocation
policies under this section, it shall consider whether to adopt
transition procedures that would treat people on the national list and
awaiting transplantation prior to the adoption or effective date of the
revised policies no less favorably than they would have been treated
under the previous policies. The transition procedures shall be
transmitted to the Secretary for review together with the revised
allocation policies.
(ii) Special rule for initial revision of liver allocation
policies. When the OPTN transmits to the Secretary its initial revision
of the liver allocation policies, as directed by paragraph (c)(2) of
this section, it shall include transition procedures that, to the
extent feasible, treat each individual on the national list and
awaiting transplantation on April 2, 1998 no less favorably than he or
she would have been treated had the revised liver allocation policies
not become effective. These transition procedures may be limited in
duration or applied only to individuals with greater than average
medical urgency if this would significantly improve administration of
the list or if such limitations would be applied only after
accommodating a substantial preponderance of those disadvantaged by the
change in the policies.
(b) Secretarial review of policies and performance Indicators. The
OPTN's transmittal to the Secretary of proposed allocation policies and
performance indicators shall include such supporting material,
including the results of model-based computer simulations, as the
Secretary may require to assess the likely effects of policy changes
and as are necessary to demonstrate that the proposed policies comply
with the performance indicators and transition procedures of paragraph
(a) of this section.
(c) Deadlines for initial reviews. (1) The OPTN shall conduct an
initial review of existing allocation policies and, except as provided
in paragraph (c)(2) of this section, no later than July 1, 1999
transmit initial revised policies to meet the requirements of
Sec. 121.8 (a), together with supporting documentation to the Secretary
for review in accordance with Sec. 121.4.
(2) No later than August 31, 1998 the OPTN shall transmit revised
policies and supporting documentation for liver allocation to meet the
requirements of Sec. 121.8 (a) to the Secretary for review in
accordance with Sec. 121.4. The OPTN may transmit these materials
without seeking further public comment under Sec. 121.4(b) or (c).
(d) Variances. The OPTN may develop experimental policies that test
methods of improving allocation. All such experimental policies shall
be accompanied by a research design and include data collection and
analysis plans. Such variances shall be time limited. Entities or
individuals objecting to variances may appeal to the Secretary under
the procedures of Sec. 121.4.
(e) Directed donation. Nothing in this section shall prohibit the
allocation of an organ to a recipient named by those authorized to make
the donation.
Sec. 121.9 Designated transplant program requirements.
(a) To receive organs for transplantation, a transplant program in
a hospital that is a member of the OPTN shall abide by these rules and
shall:
(1) Be a transplant program approved by the Secretary for
reimbursement under Medicare and Medicaid; or
(2) Be an organ transplant program which has adequate resources to
provide transplant services to its patients and agrees promptly to
notify the OPTN and patients awaiting transplants if it becomes
inactive and which:
(i) Has letters of agreement or contracts with an OPO;
(ii) Has on site a transplant surgeon qualified in accordance with
policies developed under Sec. 121.4;
(iii) Has on site a transplant physician qualified in accordance
with policies developed under Sec. 121.4;
(iv) Has available operating and recovery room resources, intensive
care resources and surgical beds and transplant program personnel;
(v) Shows evidence of collaborative involvement with experts in the
fields of radiology, infectious disease, pathology, immunology,
anesthesiology, physical therapy and rehabilitation medicine,
histocompatibility, and immunogenetics and, as appropriate, hepatology,
pediatrics, nephrology with dialysis capability, and pulmonary medicine
with respiratory therapy support;
(vi) Has immediate access to microbiology, clinical chemistry,
histocompatibility testing, radiology and blood banking services, as
well as the capacity to monitor treatment with immunosuppressive drugs;
and
(vii) Makes available psychiatric and social support services for
transplant candidates, transplant recipients and their families; or
(3) Be a transplant program in a Department of Veterans Affairs
hospital which is a Dean's Committee hospital which shares a common
university-based transplant team of a transplant program which meets
the requirements of Sec. 121.9(a) (1) or (2).
(b) To apply to be a designated transplant program, transplant
programs shall provide to the OPTN such documents as the OPTN may
require which show that they meet the requirements of Sec. 121.9(a)
(1), (2), or (3).
(c) The OPTN shall, within 90 days, accept or reject applications
to be a designated transplant program.
(d) Applicants rejected for designation may appeal to the
Secretary. Appeals shall be submitted in writing within 30 days of
rejection of the application. The Secretary may:
(1) Deny the appeal; or
(2) Direct the OPTN to take action consistent with the Secretary's
response to the appeal.
Sec. 121.10 Reviews, evaluation, and enforcement.
(a) Review and evaluation by the Secretary. The Secretary or her/
his designee may perform any reviews and evaluations of member OPOs and
transplant programs which the Secretary deems necessary to carry out
her/his responsibilities under the Public Health Service Act and the
Social Security Act.
(b) Review and evaluation by the OPTN. (1) The OPTN shall design
appropriate plans and procedures, including survey instruments, a peer
review process, and data systems, for purposes of:
(i) Reviewing applications submitted under Sec. 121.3(c) for
membership in the OPTN;
(ii) Reviewing applications submitted under Sec. 121.9(b) to be a
designated transplant program; and
(iii) Conducting ongoing and periodic reviews and evaluations of
each member OPO and transplant hospital for compliance with these rules
and OPTN policies.
(2) Upon the approval of the Secretary, the OPTN shall furnish
review plans and procedures, including survey instruments and a
description of data systems, to each member OPO and transplant
hospital. The OPTN shall furnish any revisions of these documents to
member OPOs and hospitals, after approval by the Secretary, prior to
their implementation.
(3) At the request of the Secretary, the OPTN shall conduct special
reviews of OPOs and transplant programs, where the Secretary has reason
to believe that such entities may not be in compliance with these rules
or OPTN policies or may be acting in a manner which poses a risk to the
health of patients or to public safety. The OPTN shall conduct these
reviews in accordance with such schedules as the Secretary specifies
and
[[Page 16337]]
shall make periodic reports to the Secretary of progress on such
reviews and on other reviews conducted under the requirements of this
paragraph.
(4) The OPTN shall notify the Secretary in a manner prescribed by
the Secretary within 3 days of all committee and Board of Directors
meetings in which transplant hospital and OPO compliance with these
regulations or OPTN policies is considered.
(c) Enforcement of OPTN rules. (1) OPTN recommendations. The Board
of Directors shall advise the Secretary of the results of any reviews
and evaluations conducted under paragraph (b)(1)(iii) or paragraph
(b)(3) of this section which, in the opinion of the Board, indicate
noncompliance with these rules or OPTN policies, or indicate a risk to
the health of patients or to the public safety, and shall provide any
recommendations for appropriate action by the Secretary. Appropriate
action may include removal of designation as a transplant program under
Sec. 121.9, termination of a transplant hospital's participation in
Medicare or Medicaid, termination of a transplant hospital's
reimbursement under Medicare and Medicaid, or termination of an OPO's
reimbursement under Medicare and Medicaid, if the noncompliance is with
a policy designated by the Secretary as covered by section 1138 of the
Social Security Act.
(2) Secretary's action on recommendations. Upon the Secretary's
review of the Board of Directors' recommendations, the Secretary may:
(i) Request further information from the Board of Directors or the
alleged violator, or both;
(ii) Decline to accept the recommendation;
(iii) Accept the recommendation, and notify the alleged violator of
the Secretary's decision; or
(iv) Take such other action as the Secretary deems necessary.
Sec. 121.11 Record maintenance and reporting requirements.
(a) Record maintenance. Records shall be maintained and made
available subject to OPTN policies and applicable limitations based on
personal privacy as follows:
(1) The OPTN and the Scientific Registry, as appropriate, shall:
(i) Maintain and operate an automated system for managing
information about transplant candidates, transplant recipients, and
organ donors, including a computerized national list of individuals
waiting for transplants;
(ii) Maintain records of all transplant candidates, all organ
donors and all transplant recipients;
(iii) Operate, maintain, receive, publish, and transmit such
records and information electronically, to the extent feasible, except
when hard copy is requested; and
(iv) In making information available, provide manuals, forms, flow
charts, operating instructions, or other explanatory materials as
necessary to understand, interpret, and use the information accurately
and efficiently.
(2) Organ procurement organizations and transplant programs. (i)
Maintenance of records. All OPOs and transplant programs shall maintain
such records pertaining to each potential donor identified, each organ
retrieved, each recipient transplanted and such other transplantation-
related matters as the Secretary deems necessary to carry out her/his
responsibilities under the Act. The OPO or transplant program shall
maintain these records for seven years.
(ii) Access to facilities and records. OPOs and transplant
hospitals shall permit the Secretary and the Comptroller General, or
their designees, to inspect facilities and records pertaining to any
aspect of services performed related to organ donation and
transplantation.
(b) Reporting requirements. (1) The OPTN and the Scientific
Registry, as appropriate, shall:
(i) In addition to special reports which the Secretary may require,
submit to the Secretary a report not less than once every fiscal year
on a schedule prescribed by the Secretary. The report shall include the
following information in a form prescribed by the Secretary:
(A) Information that the Secretary prescribes as necessary to
assess the effectiveness of the Nation's organ donation, procurement
and transplantation system;
(B) Information that the Secretary deems necessary for the report
to Congress required by Section 376 of the Act; and,
(C) Any other information that the Secretary prescribes.
(ii) Provide to the Scientific Registry data on transplant
candidates and recipients, and other information that the Secretary
deems appropriate. The information shall be provided in the form and on
the schedule prescribed by the Secretary;
(iii) Provide to the Secretary any data that the Secretary
requests;
(iv) Make available to the public timely and accurate program-
specific information on the performance of transplant programs. This
shall include free dissemination over the Internet, and shall be
presented, explained, and organized as necessary to understand,
interpret, and use the information accurately and efficiently. These
data shall be updated no less frequently than every six months and
shall include three month, one year, three year and five year graft and
patient survival rates, both actual and statistically expected, and
shall be presented no more than six months later than the period to
which they apply. Data presented shall include confidence intervals or
other measures that provide information on the extent to which chance
may influence transplant program-specific results. Such data shall also
include such other cost or performance information as the Secretary may
specify, including but not limited to transplant program-specific
information on waiting time within medical status, organ wastage, and
refusal of organ offers. These data shall also be presented no more
than six months later than the period to which they apply;
(v) Respond to reasonable requests from the public for data needed
for bona fide research or analysis purposes, to the extent that the
OPTN's or Scientific Registry's resources permit, or as directed by the
Secretary. The OPTN or the Scientific Registry may impose reasonable
charges for the separable costs of responding to such requests.
Patient-identified data may be made available to bona fide researchers
upon a showing that the research design requires such data for matching
or other purposes, and that appropriate confidentiality protections,
including destruction of patient identifiers upon completion of
matching, will be followed. All requests shall be processed
expeditiously, with data normally made available within 30 days from
the date of request;
(vi) Respond to reasonable requests from the public for data needed
to assess the performance of the OPTN or Scientific Registry, to assess
individual transplant programs, or for other purposes. The OPTN or
Scientific Registry may impose charges for the separable costs of
responding to such requests. An estimate of such charges shall be
provided to the requester before processing the request. All requests
should be processed expeditiously, with data normally made available
within 30 days from the date of request; and
(vii) Provide data to an OPTN member, without charge, that has been
assembled, stored, or transformed from data originally supplied by that
member.
(2) An organ procurement organization or transplant hospital shall,
as specified from time to time by the Secretary, submit to the OPTN, to
the Scientific Registry, as appropriate, and
[[Page 16338]]
to the Secretary information regarding transplantation candidates,
transplant recipients, donors of organs, transplant program
performance, and other information that the Secretary deems
appropriate. Such information shall be in the form required and shall
be submitted in accordance with the schedule prescribed. No
restrictions on subsequent redisclosure may be imposed by any organ
procurement organization or transplant hospital.
(c) Public access to data. The Secretary may release to the public
information collected under this section when the Secretary determines
that the public interest will be served by such release. The
information which may be released includes, but is not limited to,
information on the comparative costs and patient outcomes at each
transplant program affiliated with the OPTN, transplant program
personnel, information regarding instances in which transplant programs
refuse offers of organs to their patients, information regarding
characteristics of individual transplant programs, information
regarding waiting time at individual transplant programs, and such
other data as the Secretary determines will provide information to
patients, their families, and their physicians that will assist them in
making decisions regarding transplantation.
Sec. 121.12 Preemption.
No State or local governing entity shall establish or continue in
effect any law, rule, regulation, or other requirement that would
restrict in any way the ability of any transplant hospital, OPO, or
other party to comply with organ allocation policies of the OPTN or
other policies of the OPTN that have been approved by the Secretary
under this part.
[FR Doc. 98-8191 Filed 3-26-98; 8:45 am]
BILLING CODE 4160-15-P
